Drug Delivery Systems for Metabolic Disorders

Front Cover
Drug Delivery Systems for Metabolic Disorders
Copyright Page
Contents
List of contributors
About the editors
1 Introduction to metabolic disorders
1.1 Metabolic disorders due to deregulation of protein/amino acid metabolism
1.1.1 Protein metabolism disorders
1.1.1.1 Amyloidosis
1.1.1.2 Kwashiorkor
1.1.1.3 Porphyria
1.1.1.4 Erythropoietic uroporphyrin
1.1.2 Amino acid metabolism disorders
1.1.2.1 Phenylketonuria
1.1.2.2 Tyrosinemia type II
1.1.2.3 Tyrosinemia type III
1.1.2.4 Alkaptonuria
1.1.2.5 Tyrosinemia type I
1.1.2.6 Maple syrup urine disease
1.2 Metabolic disorders due to deregulation of lipid metabolism
1.2.1 Atherosclerosis
1.2.2 Cerebrotendinous xanthomatosis
1.2.3 Sitosterolemia
1.2.4 Gaucher disease
1.2.5 Tay-Sachs disease
1.2.6 Niemann pick disease
1.2.7 Letterer-Siwe disease
1.3 Metabolic disorder due to deregulation in carbohydrate metabolism
1.3.1 Galactosemia
1.3.2 Hereditary fructose intolerance
1.3.3 Fructose 1,6-diphosphatase deficiency
1.3.4 Glycogen storage disorders
1.3.5 Hurler and Scheie syndrome
1.3.6 Hunter syndrome
1.3.7 Sanfilippo syndrome
1.4 Metabolic disorder due to disturbance in hormone metabolism
1.4.1 Hypoglycemia
1.4.2 Diabetes (hyperglycemia)
1.4.3 Hypopituitarism and hyperpituitarism
1.4.3.1 Hypopituitarism (panhypopituitarism)
1.4.4 Sheehan syndrome
1.4.5 Pituitary apoplexy
1.4.6 Hyperpituitarism
1.4.7 Hypoparathyroidism
1.4.8 Hyperparathyroidism
1.4.9 Pseudohypoparathyroidism
1.4.10 Hyperthyroidism
1.4.11 Hypothyroidism
1.5 Metabolic disorder due to deregulation in lysosomal storage disorders
1.5.1 Schindler disease
1.5.2 Faber disease
1.5.3 Sandhoff diseases
1.5.4 Pycnodysostosis
1.6 Metabolic disorder due to deregulation in mitochondrial disorders
1.6.1 Wilson disease
1.6.2 Pearson marrow syndrome
1.6.3 Leigh syndrome
1.6.4 Alpers disease
1.6.5 Batten disease
References
Further reading
2 Cellular and molecular mechanisms involved in metabolic disorders
2.1 Introduction
2.2 Alterations in single-minded protein-1: downstream mediator of leptin-melanocortin pathway
2.3 Link between osteocalcin deficiency and insulin resistance
2.4 Aberrations in the JAK-STAT pathway
2.5 Alteration in AMPK activity: insulin resistance and cardiovascular problems
2.6 Conclusion
Acknowledgments
References
3 Current practices in drug delivery for metabolic disorders
3.1 Introduction
3.2 Oral drug delivery approaches
3.3 Site-specific drug delivery approaches
3.4 Targeted drug delivery
3.5 Intelligent drug delivery
3.6 Conclusion
Acknowledgments
References
4 Recent developments in the treatment of amyloidosis
4.1 Introduction
4.2 The history of amyloidosis
4.3 Amyloid fibril
4.3.1 Amyloid fibrillogenesis
4.3.2 Pathogenicity of amyloid fibril
4.3.3 Classification of amyloidosis
4.3.3.1 Localized amyloidosis
4.3.3.2 Systematic amyloidosis
4.3.3.3 AL amyloidosis
4.3.3.4 AA Amyloidosis
4.3.3.5 Aß2M Amyloidosis
4.3.3.6 ATTRwt Amyloidosis
4.3.4 Hereditary systemic amyloidosis
4.3.5 Methods used for diagnosis of amyloidosis
4.3.6 Treatment of amyloidosis
4.3.6.1 AL Amyloidosis
4.3.6.2 ATTRm amyloidosis
4.3.6.3 AA Amyloidosis
4.4 Conclusion
Acknowledgments
References
5 Advanced drug delivery systems targeting to improve therapeutic outcomes in porphyria
5.1 Introduction
5.2 Prevalence
5.3 Pathophysiology
5.3.1 5-ALA- dehydratase-deficient porphyria
5.3.2 Acute intermittent porphyria
5.3.3 Congenital erythropoietic porphyria
5.3.4 Porphyria cutanea tarda and hepatoerythropoietic porphyria
5.3.5 Hereditary coproporphyria
5.3.6 Variegate porphyria
5.3.7 Erythropoietic protoporphyria
5.3.8 X-linked protoporphyria
5.4 Symptoms and diagnosis
5.4.1 5-ALA- dehydratase-deficient porphyria
5.4.2 Acute intermittent porphyria
5.4.3 Porphyria cutanea tarda and hepatoerythropoietic porphyria
5.4.4 Hereditary coproporphyria
5.4.5 Variegate porphyria
5.4.6 Congenital erythropoietic porphyria
5.4.7 Erythropoietic protoporphyria and X-linked protoporphyria
5.5 Treatment strategies
5.5.1 Acute intermittent porphyria
5.5.2 5-ALA- dehydratase-deficient porphyria
5.5.3 Porphyria cutanea tarda and hepatoerythropoietic porphyria
5.5.4 Hereditary coproporphyria
5.5.5 Variegate porphyria
5.5.6 Congenital erythropoietic porphyria
5.5.7 Erythropoietic protoporphyria and X-linked protoporphyria
5.6 Overview of treatment drugs and current treatment problems
5.6.1 Intravenous hemin (panhematin)
5.6.2 Chloroquine and hydroxychloroquine
5.6.3 Afamelanotide
5.6.4 Beta-carotene
5.6.5 Givosiran (GIVLAARI)
5.7 Novel therapies
5.8 Summary and conclusion
References
6 Advanced drug delivery systems targeting kwashiorkor involving a disturbance in protein metabolism
6.1 Introduction
6.2 Physiological changes associated with kwashiorkor disease
6.3 Biochemical manifestations and metabolic disorders associated with kwashiorkor disease
6.4 Signs, symptoms, and clinical manifestations in kwashiorkor
6.5 Current treatment of kwashiorkor
6.6 Metabolic disturbances associated with kwashiorkor
6.7 Role of nanotechnology in drug-delivery
6.8 Different types of drug delivery vehicles
6.9 Advanced drug delivery system targeting kwashiorkor
6.10 Conclusion
References
7 Advanced drug delivery systems targeting metabolic disorders: erythropoietic protoporphyria
7.1 Introduction
7.2 Etiology
7.3 Clinical features
7.3.1 Dermatologic involvement
7.3.2 Hematological involvement
7.4 Diagnosis
7.5 Pathophysiology
7.6 Genotype-phenotype correlations
7.7 Current and emerging therapies
7.7.1 Modulation of iron supply
7.7.2 Antisense oligonucleotide therapy
7.7.3 Increased light tolerance with antioxidants or stimulators of skin melanin synthesis
7.7.4 Surgery
7.7.5 Consultations
7.8 Conclusion and future perspective
References
8 Combating atherosclerosis with nanodrug delivery approaches: from bench side to commercialization
8.1 Introduction
8.2 Atherosclerosis
8.2.1 Etiology
8.2.2 Pathogenesis
8.2.3 Clinical effects
8.2.4 Pharma-armamentarium and mechanistic insights
8.3 Nanoformulations for the treatment of atherosclerosis with implementation of quality by design
8.3.1 Nanoparticles
8.3.1.1 Implementation of the concept of quality by design for nanoformulations
8.3.2 Liposomes
8.3.2.1 Liposomes in treatment of atherosclerosis
8.3.2.2 Implementation of the concept of QbD in the formulation of liposomes
8.4 Dendrimers
8.4.1 Implementation of concept QbD during the formulation of dendrimer
8.5 Challenges in translating nanodrug delivery systems from bench to bedside
8.5.1 Scalability
8.5.2 Safety
8.5.3 Process optimization
8.5.4 Stability of the product
8.5.5 GRAS status of the material
8.5.6 Regulatory clearance
8.5.7 Nanoparticles
8.5.8 Liposomes
8.5.9 Dendrimer
8.6 Conclusion
References
9 Advanced drug delivery systems in the management of Gaucher disease
9.1 History
9.2 Epidemiology
9.3 Etiology
9.4 Pathogenesis
9.5 Clinical classification of Gaucher disease
9.6 Management of Gaucher disease
9.6.1 Baseline management
9.6.2 Disease-specific management
9.6.2.1 Enzyme replacement therapies
9.6.2.1.1 Liposome encapsulated GCase
9.6.2.1.2 RBCs encapsulated GCase
9.6.2.1.3 Micro-carriers loaded with GCase
9.6.2.2 Substrate reduction therapies
9.6.3 Other specific treatments
9.6.3.1 Pharmacological chaperone therapies
9.6.3.1.1 Iminosugars
9.6.3.1.2 Ambroxol
9.6.3.1.3 Noninhibitory chaperone
9.6.3.2 Histone deacetylase inhibitors
9.6.3.3 Gene therapy
9.6.3.4 Bone marrow transplant and total joint replacement
9.6.3.5 Splenectomy
9.7 Conclusion
References
10 Advanced drug delivery systems focusing the metabolic disorders due to deregulation of lipid metabolism: Niemann Pick Di...
10.1 Introduction
10.1.1 Lipid metabolic disorder
10.2 Neimann’s Pick disease
10.2.1 Common symptoms of Neimann’s disease
10.3 Etiology
10.3.1 The molecular biology underlying NP-C pathophysiology
10.3.2 Lipid trafficking and NPC1: cholesterol transport
10.3.3 NPC protein function: NPC1 & NPC2
10.3.4 Diagnosis
10.3.5 History taking and clinical examination
10.3.6 Laboratory findings
10.3.7 Genetic testing
10.3.8 Pathophysiology of sphingomyelinase deficiency Niemann’s pick disease
10.3.9 Niemann’s Pick disease type A
10.3.10 Niemann’s Pick disease type B
10.3.11 Niemann’s Pick disease type C
10.3.12 Regular laboratory testing
10.3.13 Diagnostic biomarkers
10.3.14 Plasma oxysterols
10.3.15 Plasma lysosphingolipids
10.3.16 Management of NPDs
10.3.17 Letterer-Siwe disease
10.3.18 Sign and symptoms
10.3.19 Pathophysiology of Letterer Siwe disease
10.4 Stage 1: early lesion’s phase
10.5 Stage 2: fibrous phase
10.6 Stage 3: extra lesion phase
10.7 Diagnosis Letterer Siwe disease
10.7.1 Physical diagnosis
10.7.2 Laboratory diagnosis
10.7.3 Epidemiology
10.7.4 Treatment
10.7.5 Novel drug delivery systems for treatment of Niemann’s Pick disease and Letterer-Siwe disease
10.7.6 Resealed erythrocytes
10.7.7 Liposomes
10.7.8 Microcapsules
10.7.9 Nanocarriers
10.8 Prospects
10.9 Conclusion
References
11 Genetic disease and Niemann-Pick disorders: novel treatments and drug delivery systems
11.1 Introduction to genetic disorders
11.2 Classification of inherited metabolic disorders
11.3 Physiology of lipid metabolism and diseases associated with lipid metabolism
11.4 Essential aspects of Niemann-Pick disease: prevalence, pathophysiology, diagnosis, and symptoms
11.5 Current therapeutic strategies to treat Niemann-Pick disease
11.6 Problems associated with the current therapeutic strategies to treat Niemann-Pick disease
11.7 Possible novel drug delivery systems to improve therapeutic efficacy in Niemann-Pick disease
References
12 Novel 3D printing drug delivery system for the prevention and treatment of Atherosclerosis
12.1 Importance of 3D printing drug delivery systems
12.2 Pathophysiology of atherosclerosis
12.3 Drugs used for the prevention and treatment of atherosclerosis
12.4 Problems with current oral drug delivery
12.5 Customized 3D printed dosage forms for atherosclerosis and associated diseases
12.6 Conclusion
References
13 Advanced drug delivery systems to treat Huntington’s disease: challenges and opportunities
13.1 Introduction
13.2 Global prevalence and economic burden
13.3 Pathophysiology
13.3.1 Genetic factors
13.3.2 Mitochondrial dysfunction
13.3.3 Oxidative stress
13.3.4 Neurodegeneration
13.4 Pharmacological management of Huntington’s disease
13.5 Herbal therapy
13.6 Challenges associated with conventional therapy and the need for advanced drug delivery systems
13.6.1 Nanoemulsions
13.6.1.1 Method of preparation of nanoemulsions
13.6.2 Self-emulsifying drug delivery system
13.6.2.1 Advantages of self-emulsifying drug delivery system over other drug delivery systems
13.6.2.2 Mechanism of self-emulsification, dissolution enhancement, and drug transport by self-emulsifying drug delivery system
13.6.3 Liposomes
13.6.4 Polymeric micelles
13.6.5 Nanostructured lipid carriers
13.6.6 Solid lipid nanoparticles
13.7 Conclusion and perspectives
References
14 Advanced drug delivery systems for targeting obesity
14.1 Introduction
14.2 Reason for weight gain
14.3 Obesity management
14.4 Conventional anti-obesity treatments
14.5 Advanced drug delivery systems
14.5.1 Nanoparticle
14.5.2 Metallic nanoparticles
14.5.3 Solid lipid nanoparticles
14.5.4 Liposomes
14.5.5 Microneedles
14.5.6 Nanoemulsion
14.5.7 Gene-based therapy
14.5.8 Interleukins
14.6 Conclusion and future prospectus
References
15 Advanced drug delivery systems for treatment of diabetes mellitus
15.1 Introduction
15.2 Antidiabetic medication-related complications
15.3 Possibilities provided by the various Novel drug delivery systems
15.4 Development and designing of various advanced drug delivery systems for diabetes treatment
15.4.1 Particulate transport system
15.4.2 Microparticulate systems
15.4.3 Nanoparticulate systems
15.4.4 Vesicular system
15.4.4.1 Liposomes
15.4.4.2 Niosomes
15.4.5 Transdermal drug delivery systems
15.4.5.1 Employment of microneedle in patch
15.4.5.2 Gelation and hydroxyapatite fabricated bioceramic composite microneedle
15.4.5.3 Double-layered, bullet-shaped microneedle with swellable tips patch
15.4.5.4 Biodegradable alginate and hyaluronate polymer microneedle patch
15.4.5.5 Poly- γ-glutamic acid microneedles with supporting structure
15.4.5.6 Alginate and maltose microneedle patch
15.4.6 Transdermal delivery systems incorporated with biosensor
15.4.6.1 Swellable microneedle patch in interstitial fluid extraction for glucose metabolic analysis
15.4.6.2 Patch-type 3D stainless steel microneedle array enzyme-free glucose biosensor
15.4.6.3 A composite nanostructured surface electrochemical glucose sensor
15.4.6.4 The ultra-miniaturization planar amperometric glucose sensor
15.4.6.5 Lab-on-chip with triboelectric liquid volume sensor
15.4.7 Point of care therapy
15.4.7.1 H2O2-responsive polymeric vesicle with microneedle
15.4.7.2 Insulin-loaded and H2O2-responsive mesoporous silica nanoparticle integrated microneedle patch
15.4.7.3 Sweat-based electrochemical patch with thermoresponsive microneedle
15.4.7.4 Hypoxia and H2O2 dual-sensitive polymersome based vesicle smart insulin patch
15.4.8 Employment of technologies for carrier, entrapment, penetration, and release
15.4.8.1 Permeation enhancement via proniosomal gel entrapment
15.4.8.2 Proniosome carbopol-based transgel system
15.4.8.3 Transferosomal gel with the chemical “iodophor”
15.4.8.4 Microemulsion gel
15.4.8.5 Transdermal nanoemulsion encapsulation
15.4.8.6 Nanostructured lipid carriers transdermal system
15.4.8.7 Hyaluronic acid encapsulated CuS gel-mediated near-infrared laser nanosystem
15.4.8.8 Choline and geranate (CAGE) deep eutectic solvent transdermal delivery vehicle
15.4.8.9 Amidated pectin hydrogel matrix patch
15.4.8.10 HPMC and PVA based transdermal patch
15.5 Prospects and conclusion
References
16 Advanced drug delivery system in the treatment of hyperglycemia and hypoglycemia
16.1 Introduction
16.1.1 Carbohydrate metabolism disorders
16.1.2 Galactose and fructose metabolism disorders
16.1.3 Glycogen storage disorders
16.1.4 Glucose transport disorder
16.1.5 Congenital disorders of glycosylation
16.1.6 Glucose metabolism disorders
16.1.6.1 Type 1 diabetes mellitus
16.1.6.2 Type 2 diabetes mellitus
16.1.6.3 Gestational diabetes mellitus
16.2 Conventional treatment for glucose metabolism disorders
16.2.1 Insulin therapies
16.2.2 Herbal treatment for diabetes mellitus
16.3 Alternative approaches to diabetes
16.3.1 Dietary fibers
16.3.2 Minerals used in the treatment of hyperglycemia
16.4 Nanomaterials for treatment of diabetes
16.4.1 Oral administration
16.4.2 Inhalations
16.4.3 Nano-pumps
16.4.4 Artificial pancreas
16.5 Advances in insulin delivery
16.6 Conclusion and future perspectives
References
17 Emerging drug delivery in the treatment of hyperpituitarism and hypopituitarism
17.1 Introduction
17.1.1 Pituitary gland (hypophysis)
17.1.2 Hormones of pituitary gland
17.1.3 Human growth hormone or somatotropin
17.1.4 Thyroid-stimulating hormone or thyrotropin
17.1.5 Prolactin or mamotrophin hormone or luteotrophic hormone
17.1.6 Follicle-stimulating hormone
17.1.7 Luteinizing hormone
17.1.8 Adrenocorticotropic hormone
17.1.9 Melanocyte-stimulating hormone
17.1.10 Antiduretic hormone or vasopressin or pitressin
17.1.11 Oxytocin (OT, Pitocin)
17.2 Treatment of hypopituitarism and hyperpituitarism
17.2.1 Treatment of hypopituitarism
17.2.1.1 Adrenocorticotropic hormone replacement therapy
17.2.1.2 Thyroid-stimulating hormone deficiency replacement therapy
17.2.1.3 Gonadotropin deficiency treatment
17.2.1.4 Growth hormone deficiency treatment
17.2.2 Treatment of hyperpituitarism
17.3 Conclusion and future perspective
References
18 Advance drug delivery systems targeting hypoparathyroidism and hyperparathyroidism
18.1 Introduction
18.1.1 Parathyroidism
18.1.1.1 Hyperparathyroidism
18.1.1.2 Hypoparathyroidism
18.2 Pathophysiology
18.2.1 Pathophysiology of hyperparathyroidism
18.2.2 Pathophysiology of hypoparathyroidism
18.3 Clinical manifestations
18.4 Current treatments for hypoparathyroidism and hyperparathyroidism
18.4.1 Advance drug delivery therapy for hypoparathyroidism
18.4.2 Advance drug delivery therapy for hyperparathyroidism
18.5 Conclusion
References
19 An update on the development of advanced drug delivery systems for the treatment of hyperthyroidism
19.1 Introduction
19.2 Epidemiology of hyperthyroidism
19.3 Common etiology and pathogenesis of hyperthyroidism
19.3.1 Grave’s disease
19.3.2 Toxic nodular disease
19.3.3 Thyroiditis
19.3.4 Hyperthyroidism due to drug interactions
19.3.5 Hyperthyroidism due to iodine
19.4 Diagnosis of hyperthyroidism
19.5 Advancement in drug delivery systems/techniques for the management of hyperthyroidism
19.5.1 Methimazole
19.5.2 Propylthiouracil
19.5.3 Carbimazole
19.5.4 Iodine containing compounds
19.5.5 Potassium perchlorate
19.5.6 Beta-blockers
19.5.7 Lithium carbonate
19.5.8 Glucocorticoids
19.6 Potential future therapies
19.6.1 Immunotherapy
19.6.2 Rituximab
19.6.3 CFZ533
19.6.4 ATX-GD-59
19.6.5 K1–70
19.6.6 Ultrasound based therapies
19.6.7 Radiofrequency ablation
19.7 Nanoparticles: promising auxiliary agents for therapy of hyperthyroidism and associated diseases
19.7.1 Inorganic nanoparticles: carbon nanoparticles
19.7.2 Inorganic Nanoparticles: metal-containing nanoparticles
19.7.3 Inorganic and hydrid nanoparticles: mesoporous silica and organic mesoporous silica nanoparticles
19.7.4 Organic nanoparticles: lipid nanoparticles
19.7.5 Nanoparticles in clinical trials
19.8 Conclusions and future perspectives
References
20 Advanced drug delivery systems involving lysosomal storage disorders for Schinder disease and other disorders
20.1 Introduction to lysosomal storage disorders
20.2 Potential targets for lysosomal disorders
20.2.1 Targeting the affected genes
20.2.2 Targeting the lipid storage
20.2.3 Targeting lysosomes and autophagy
20.2.4 Targeting mitochondria and oxidative stress
20.2.5 Targeting calcium imbalance
20.2.6 Targeting synaptic impairment and neuron excitability
20.2.7 Targeting inflammation
20.2.8 Targeting hypomyelination
20.3 Potential strategies using advanced delivery systems for lysosomal storage diseases
20.3.1 Enzyme replacement therapy
20.3.2 Bone marrow transplantation
20.3.3 Small molecule therapies
20.3.3.1 Use of molecular chaperones
20.3.3.2 Proteostasis regulators
20.3.3.3 Substrate reduction therapy
20.3.3.4 Small molecules targeting nonsense mutations
20.3.4 Hematopoietic stem cell therapy
20.3.5 CSF-delivery of enzyme replacement therapy agents
20.3.6 Nanocarrier delivery therapies
20.4 Attempts in treatment of lysosomal storage diseases by utilization of gene therapy
20.4.1 Concept of gene therapy for lysosomal storage disorder
20.4.1.1 Viral vectors for gene therapy
20.4.1.1.1 Retroviruses
20.4.1.1.2 Lentiviruses
20.4.1.1.3 Adenoviruses
20.4.1.1.4 Adeno-associated viruses
20.4.2 Systemic gene therapy
20.4.3 CNS—directed gene therapy
20.4.4 Future-generation gene therapy strategies
20.5 Current novel lysosomal storage disorder therapies in clinical trials
Acknowledgments
References
21 Advanced drug delivery systems involving lysosomal storage disorders for Fabry disease
21.1 Introduction
21.2 Classification and clinical manifestations
21.3 Diagnosis
21.4 Treatments
21.4.1 Enzyme replacement therapy
21.4.2 Pharmacological chaperones
21.5 New approaches for the treatment of Fabry disease
21.5.1 Second generation enzyme therapies
21.5.1.1 Pegunigalsidase-alpha (PRX-120)
21.5.1.2 Moss-α-GAL A (Moss derived α GAL A)
21.5.1.3 Modified α-N-Acetylgalactosaminidase (NAGA)
21.5.2 Substrate reduction therapy
21.5.3 Gene therapy
21.5.3.1 Ex-vivo gene therapy
21.5.3.2 In-vivo gene therapy
21.5.4 mRNA therapy
21.5.4.1 Pros and cons of the emerging therapies
21.6 Advanced drug delivery systems in Fabry disease
21.6.1 Liposomes
21.6.2 Polystyrene capsules
21.6.3 Polyelectrolyte complexes
21.6.4 Solid-lipid nanoparticles
21.6.5 Extracellular vesicles
21.7 Conclusion
References
22 Drug delivery systems in Krabbe disease—present and prospective approaches
22.1 Introduction
22.2 Genetic background
22.3 Pathophysiology
22.3.1 Sulfatide insufficiency
22.3.2 Galactocerebroside β-galactosidase deficiency
22.3.3 The psychosine hypothesis
22.3.4 Saposin A deficiency
22.4 Current approaches and challenges
22.4.1 Single modality therapies
22.4.1.1 HSCT with bone marrow transplantation
22.4.1.2 Oligodendrocyte transplantation
22.4.1.3 Neuronal and mesenchymal stem cell transplantation
22.4.1.4 Neural stem cell gene therapy
22.4.1.5 Virus mediated gene therapy
22.4.1.6 Enzyme replacement therapy
22.4.1.7 Substrate reduction therapy
22.4.1.8 Antiinflammatory approaches
22.4.1.9 Antioxidant therapy
22.4.2 Multimodality therapies
22.4.2.1 Vascular endothelial growth factor+bone marrow transplantation
22.4.2.2 L-cycloserine+bone marrow transplantation
22.4.2.3 N-Acetylcysteine+bone marrow transplantation
22.4.2.4 Enzyme replacement therapy+bone marrow transplantation
22.4.2.5 Gene therapy+bone marrow transplantation
22.4.2.6 Gene therapy+substrate reduction therapy+bone marrow transplantation
22.5 Potential targets and approaches
22.5.1 Advances in substrate reduction therapy
22.5.1.1 S2O2, a novel GalCer inhibitor
22.5.1.2 A chemotherapeutic agent as a potential therapeutic agent
22.5.2 Enzyme-loaded nanoparticles
22.5.3 Pharmacological chaperons
22.5.3.1 α-Lobeline as a potential agent
22.5.3.2 3′,4′,7-trihydroxyisoflavone
22.5.3.3 N-octyl-4-epi-β-valienamine
22.5.3.4 Azasugars and iminosugars as potential candidates
22.5.4 Quantum dots as nano-vehicle
22.6 Conclusion
References
23 Strategies to combat Tay-Sachs disease
23.1 Introduction
23.2 Overview of Tay-Sachs disease
23.2.1 Pathophysiology of gangliosidosis and HexA synthesis
23.3 Treatment strategies and management
23.3.1 Substrate reduction therapy
23.3.1.1 Application of substrate reduction therapy in Tay-Sachs disease
23.3.2 Bone marrow transplantation
23.3.2.1 Application of bone marrow transplantation in Tay-Sachs disease
23.3.3 Enzyme replacement therapy
23.3.3.1 Application of enzyme replacement therapy for Tay-Sachs disease
23.3.4 Gene therapy
23.3.4.1 Application of gene therapy in Tay-Sachs disease
23.4 Developmental treatment for Tay-Sachs disease
23.4.1 Genetically modified multipotent cells
23.5 Conclusion
References
24 Sandhoff disease: pathology and advanced treatment strategies
24.1 Lysosomal storage disease
24.2 Sandhoff disease
24.2.1 Introduction
24.3 Etiology and pathogenesis
24.3.1 Normal function of HEXB gene
24.3.2 Mutation in the gene HEXB
24.4 Prevalence and occurrence
24.5 Clinical characteristics
24.6 Treatment
24.6.1 Substrate replacement therapy
24.6.2 Enhanced gene therapy through complimentary drug therapy
24.6.3 Suppression through bone marrow transplantation
24.7 Carriers for lysosomal drug delivery
24.7.1 Resealed erythrocytes
24.8 Liposomes
24.9 Future developments on sandhoff disease
24.10 Conclusion
References
25 Nanomaterials-based drug delivery approaches for metabolic disorders
25.1 Introduction
25.2 Routes of drug delivery for metabolic disorders
25.2.1 Drug delivery strategies
25.2.1.1 Targeted drug delivery strategies
25.2.1.2 Intelligent stimulus-responsive drug delivery strategies
25.2.2 Different drug administration routes
25.2.2.1 Oral route of drug delivery approach
25.2.2.2 Local drug delivery strategies
25.2.2.3 Transdermal drug delivery approach
25.2.2.4 Inhalation/nasal drug delivery route
25.2.2.5 Intravenous drug delivery route
25.3 Nanomaterial-based drug delivery approaches
25.3.1 Nanomaterial based drug delivery systems
25.3.1.1 Polymeric drug delivery system: micelles
25.3.1.2 Polymeric drug delivery system: dendrimers
25.3.1.3 Liposomes mediated drug delivery system
25.3.1.4 Protein-based drug delivery systems
25.3.1.5 Metal nanoparticle-based delivery systems
25.3.1.6 Composite based nanomaterials
25.3.2 Different drug delivery routes of nanocarriers
25.3.2.1 Nanomaterial-based oral-drug delivery
25.3.2.2 Nano-based transdermal drug delivery
25.3.2.3 Nano-based drug approaches for nasal route
25.3.3 Nanotoxicity
25.3.4 Blood-brain barrier and nano-based drug delivery
25.4 Applications of nano-based drug delivery systems to treat metabolic disorders
25.4.1 Treatment of diabetes
25.4.1.1 Liposomes mediated drug delivery
25.4.1.2 Niosomes-based treatment
25.4.2 Treatment of pulmonary hypertension
25.4.3 Treatment of cardiovascular diseases
25.5 Limitations of using nano-based drug delivery approach
25.6 Future prospective
25.7 Conclusion
References
26 Eye in metabolic disorders: manifestations and drug delivery systems
26.1 Introduction
26.2 Diabetic retinopathy (Dr)
26.2.1 Prevalence of diabetes
26.2.2 Types and management of Diabetic retinopathy
26.2.3 Treatment of Diabetic retinopathy
26.2.4 Drug delivery systems for managing Diabetic retinopathy
26.2.4.1 Nanotechnology for diagnosis
26.2.4.2 Nanoparticles
26.2.4.3 Magnetic nanoparticles
26.2.4.4 Liposomes
26.2.4.5 Nanomicelles
26.2.4.6 Hydrogels
26.3 Glaucoma
26.3.1 Types of glaucoma and symptoms
26.3.1.1 Management of glaucoma
26.3.1.1.1 Laser treatment aspects for glaucoma
26.3.1.1.2 Conventional surgical approaches
26.3.1.1.3 Medication-based delivery system
26.3.1.1.4 Nanoparticles
26.3.1.1.5 Polymeric nanoparticles
26.3.1.1.6 Solid lipid nanoparticles
26.3.1.1.7 Silica nanoparticles
26.3.1.1.8 Liposomes
26.3.1.1.9 Polymeric hydrogels
26.3.1.1.10 Dendrimers
26.3.1.1.11 Cubosomes
26.3.1.1.12 Transfersomes
26.4 Cataract
26.4.1 Disease and its prevalence
26.4.2 Causes of cataract
26.4.2.1 Metabolic cataract
26.4.2.1.1 Galactosemic cataract
26.4.2.1.2 Diabetic cataract
26.4.2.1.3 Hypocalcemic cataract
26.4.2.1.4 Cataracts due to error in copper metabolism
26.4.3 Management of cataract
26.4.3.1 Oxidative/antioxidative system
26.4.3.2 Aldose reductase inhibitors
26.4.3.3 Dissolution of crystalline aggregates
26.4.4 Current therapies for the treatment of cataract
26.4.4.1 Cataracts surgery
26.4.4.2 Drug delivery systems
26.4.4.2.1 Solution
26.4.4.2.2 In situ gelling system
26.4.4.2.3 Colloidal drug delivery systems
26.4.4.2.4 Liposomes
26.4.4.2.5 Micro or nano emulsions
26.5 Conclusion
References
27 Advanced drug delivery systems involving mitochondrial disorders
27.1 Introduction
27.2 Mitochondrial DNA
27.3 Clinical manifestations of mitochondrial disorders
27.4 Mitochondrial diseases
27.4.1 Alpers disease
27.4.2 Batten disease
27.4.3 Pearson marrow syndrome
27.4.4 Luft disease
27.4.5 Leigh syndrome
27.4.6 Wilson disease
27.4.6.1 Drug delivery systems for Wilson disease
27.4.6.1.1 Nanoparticles
27.4.6.1.2 Liposomes
27.5 Delivery systems for mitochondrial diseases
27.5.1 Liposomes
27.5.2 Polymeric nanoparticles
27.5.3 Dendrimers
27.6 Peptide-based mitochondrial-targeted strategy
27.7 Mitochondrial targeting sequences
27.8 Szeto–Schiller peptides
27.9 Vehicle-based mitochondrial-targeted strategy
27.10 MITO-Porter
27.11 Mesoporous silica nanoparticles
27.12 Conclusions
References
28 Applications of phytopharmaceuticals in targeting metabolic disorders
28.1 Introduction
28.2 Role of polyphenols and terpenoids in the treatment of metabolic disorders
28.2.1 Effect of polyphenols on hyperglycemia
28.2.2 Hypertension
28.2.3 Obesity
28.2.4 Dyslipidemia
28.3 Management of metabolic disorders via phytomedicinal therapy
28.3.1 Panax ginseng
28.3.2 Rhizoma coptidis
28.3.3 Momordica charantia
28.3.4 Ligustrum lucidum Ait
28.3.5 Glycyrriza uralensis Fisch
28.3.6 Hoodia gordonii
28.3.7 Crataegus laevigata
28.3.8 Vitis vinifera
28.3.9 Vaccinium angustifolium Aiton
28.3.10 Artemisia dracunculus L
28.3.11 Cassia obtusifolia L
28.3.12 Polygonatum odoratum
28.3.13 Rehmannia glutinosa
28.3.14 Cucurbita moschata
28.3.15 Aloe vera L
28.4 Future prospective of phytopharmaceuticals for metabolic disorders
28.5 Conclusion
References
29 Phytonanoparticles toward the treatment of diabetes
29.1 What is diabetes?
29.2 Classification and treatment available for diabetes
29.2.1 Type 1 diabetes
29.2.2 Type 2 diabetes
29.2.3 Genetic defects of beta-cell function
29.2.4 Gestational diabetes mellitus
29.3 Treatment available for diabetes
29.4 Nanoparticles for the treatment of diabetes
29.5 Advantages of phytoderived nanoparticles
29.6 List of nanoparticles for the treatment of diabetes
29.7 List of phytonanoparticles for the treatment of diabetes
29.7.1 Piperine
29.7.1.1 Piperine coated gold nanoparticles
29.7.1.1.1 Synthesis of Piperine coated gold nanoparticles
29.7.1.1.2 Size of nanoparticles
29.7.1.1.3 Studies on piperine coated gold nanoparticles and insulin fibrils interaction
29.7.1.1.4 Mode of action
29.7.1.2 Pioglitazone loaded Bovine Serum Albumin nanoparticles with piperine
29.7.1.2.1 Synthesis of Pioglitazone loaded Bovine Serum Albumin nanoparticles with piperine
29.7.1.2.2 Size of the nanoparticles
29.7.1.2.3 Studies on Pioglitazone loaded Bovine Serum Albumin nanoparticles with piperine
29.7.1.2.4 Mode of action
29.7.2 Lignin
29.7.2.1 Silver phenolated lignin nanoparticles
29.7.2.1.1 Synthesis of silver phenolated lignin nanoparticles
29.7.2.1.2 Size of the nanoparticles
29.7.2.1.3 Studies on silver phenolated lignin nanoparticles
29.7.2.1.4 Mode of action
29.7.3 Quercetin
29.7.3.1 Quercetin conjugated superparamagnetic iron oxide nanoparticles
29.7.3.1.1 Synthesis of Quercetin coated iron oxide nanoparticles
29.7.3.1.2 Size of the nanoparticles
29.7.3.1.3 Studies on Quercetin conjugated superparamagnetic iron oxide nanoparticles
29.7.3.1.4 Mode of action
29.7.3.2 Quercetin enveloped Soluplus/P407 micelles
29.7.3.2.1 Synthesis of Quercetin-Soluplus/P407 micelles
29.7.3.2.2 Size of the nanoparticle
29.7.3.2.3 Studies on Quercetin enveloped Soluplus/P407 micelles
29.7.3.2.4 Mode of action
29.7.4 Cellulose
29.7.4.1 AgNPs embedded in cellulose nanocrystals
29.7.4.1.1 Synthesis of AgNPs embedded in cellulose nanocrystals
29.7.4.1.2 Size of the nanoparticles
29.7.4.1.3 Studies on AgNPs embedded in cellulose nanocrystals
29.7.4.1.4 Mode of action
29.7.5 Curcumin
29.7.5.1 Nanocurcumin
29.7.5.1.1 Synthesis of nanocurcumin
29.7.5.1.2 Size of the nanoparticle
29.7.5.1.3 Studies on nanocurcumin
29.7.5.1.4 Mode of action
29.7.5.2 Curcumin-zinc oxide composite nanoparticles
29.7.5.2.1 Synthesis of Curcumin-zinc oxide composite nanoparticles
29.7.5.2.2 Size of the nanoparticles
29.7.5.2.3 Studies on Curcumin-zinc oxide composite nanoparticles
29.7.5.2.4 Mode of action
29.7.6 Berberine
29.7.6.1 O-hexadecyl-dextran entrapped berberine chloride nanoparticles
29.7.6.1.1 Synthesis of O-hexadecyl-dextran entrapped berberine chloride nanoparticles
29.7.6.1.2 Size of the nanoparticles
29.7.6.1.3 Studies on O-hexadecyl-dextran entrapped berberine chloride nanoparticles
29.7.6.1.4 Mode of action
29.7.7 Metformin
29.7.7.1 Metformin loaded chitosan nanoparticles
29.7.7.1.1 Synthesis of metformin loaded chitosan nanoparticles
29.7.7.1.2 Size of the nanoparticles
29.7.7.1.3 Studies on metformin loaded chitosan nanoparticles
29.7.7.1.4 Mode of action
29.7.7.2 Metformin-loaded alginate nanoparticles
29.7.7.2.1 Synthesis of Metformin-loaded alginate nanoparticles
29.7.7.2.2 Size of the nanoparticle
29.7.7.2.3 Studies on Metformin-loaded alginate nanoparticles
29.7.7.2.4 Mode of action
29.7.8 Lectins
29.7.8.1 Lectin-modified insulin liposomes
29.7.8.1.1 Synthesis of lectin liposomes
29.7.8.1.2 Size of the nanoparticles
29.7.8.1.3 Studies on lectin-modified insulin liposomes
29.7.8.1.4 Mode of action
29.7.9 Inulin
29.7.9.1 Nanomicelles of inulin D α-tocopherol succinate
29.7.9.1.1 Synthesis of nanomicelles of inulin D α-tocopherol succinate
29.7.9.1.2 Size of the nanoparticle
29.7.9.1.3 Studies on nanomicelles of inulin D α-tocopherol succinate
29.7.9.1.4 Mode of action
29.8 Conclusion
Acknowledgments
References
30 Role of nutraceuticals in metabolic syndrome
30.1 Introduction
30.2 Definition, pathogenesis, and pathophysiology of metabolic syndrome
30.2.1 Pathogenesis and pathophysiology of Ms
30.2.1.1 Insulin resistance
30.2.1.2 Inflammatory condition
30.2.1.2.1 Hypertension
30.3 Applications of nutraceuticals in the management of Ms
30.3.1 Quercetin
30.3.2 Curcumin
30.3.3 Cinnamon
30.3.4 Resveratrol
30.3.5 Vitamins
30.3.6 Dietary Fibers
30.3.7 Omega-3-fatty acid
30.3.7.1 Macro and trace elements
30.4 Conclusion
References
31 Latest advances of phytomedicine in drug delivery systems for targeting metabolic disorders
31.1 Introduction
31.2 Significance of phytomedicines as therapeutics
31.2.1 Phytomedicine: from ancient to modern therapy system
31.3 Pathophysiology of metabolic syndrome
31.3.1 Resistance to insulin
31.3.2 Neurohormonal stimulation
31.3.3 Inflammatory responses
31.3.3.1 TNF-alpha: a tumor necrosis factor
31.3.3.2 C-reactive protein and interleukin 6
31.4 Phytomedicines in drug delivery system
31.4.1 Administration of phytomedicines
31.4.2 Nano-based drug administration
31.4.3 Nano-based drug delivery to treat metabolic disorders
31.4.3.1 Diabetes
31.4.3.2 Obesity
31.4.3.3 Dyslipidemia
31.4.3.4 Pulmonary arterial hypertension
31.5 Future perspective
31.6 Conclusion
References
32 Clinical trials, future prospects and challenges of drug delivery in combating metabolic disorders
32.1 Challenges faced by advanced delivery systems
32.2 Future prospects
32.3 Conclusion
References
Index
Back Cover