Brings industrial practitioners and researchers together to discuss how the deeper integration of biomaterial platforms could play a significant role in enabling breakthroughs in the application of gene editing for the treatment of human disease.
Table of ContentsForeword
Preface
Acknowledgment
1. Biocompatible Hydrogels for Gene Therapy: Advancement and ApplicationsAnkita Gupta and Swatantra K.S. Kushwaha
1.1 Introduction
1.2 Hydrogels Classification
1.3 Fabrication of Hydrogels and Its Desirable Technical Features
1.4 Factors to be Tuned for Gene Encapsulation in Hydrogels
1.5 Recent Advances on Hydrogels for Gene Delivery
1.6 Conclusion
References
2. Use of Polysaccharides: Novel Delivery System for Genetic MaterialPrashant Kumar, Swatantra K.S. Kushwaha, Neelottama Kushwaha, Abhishek Singh and Surya Nath Pandey
2.1 Introduction
2.1.1 Commonly Available Polysaccharide-Based Natural Biomaterials with Their Physicochemical Properties
2.1.2 Advantages of Polysaccharides-Based Potent Biomaterials
2.1.3 Limitations
2.2 Cross-Linking Techniques for Engineering Polysaccharides-Based Biomaterials
2.2.1 Physical Cross-Linking
2.2.2 Chemical Cross-Linking
2.2.3 Ionically Cross-Linked
2.3 Approaches to Design Polysaccharide-Derived Biomaterials
2.3.1 Etherification of Polysaccharides
2.3.2 Esterification of Polysaccharide
2.3.3 Sulfation of Polysaccharides
2.3.4 Benzoylations of Polysaccharides
2.3.5 Acetylation of Polysaccharides
2.3.6 Phosphorylation of Polysaccharides
2.3.7 Amination of Polysaccharides
2.3.8 Carboxymethylation of Polysaccharides
2.3.9 Condensation with Ketones
2.4 Biomedical Applications of Polysaccharide-Derived Biomaterials
2.4.1 Polymer-Drug Conjugates
2.5 Advanced Biomaterials for Wound Dressings
2.6 Scaffolds for Tissue Engineering and Development of Bioinks for 3D Bioprinting
2.7 Recent Utilization of Polysaccharides
2.7.1 Bone/Cartilage Regeneration
2.7.2 Cardiac Regeneration
2.7.3 Neural Regeneration
2.7.4 Skin Regeneration
2.7.5 As a Hemostatic Agent
2.7.6 Stimulation of Angiogenesis
2.8 Toxicity Concerns of Polysaccharide-Derived Biomaterials
2.9 Preclinical and Clinical Studies on Gene Delivery Using Polysaccharide-Based Biomaterials
2.10 Challenges and Future Directions
2.11 Future Prospects
2.12 Conclusion
References
3. Polysaccharide-Based Biomaterials for Gene DeliveryAnkita Moharana, Abhitav Tiwari, Shalini Perada, Shivlal Yadav and Om Prakash Kumar
3.1 Background
3.2 Introduction
3.3 Gene Therapy
3.3.1 Why Does Gene Therapy Need a Carrier?
3.3.2 Translational Relevance
3.4 Gene Delivery Systems Based on Polysaccharides
3.4.1 Polysaccharide-Based Nanocarriers
3.4.2 Polysaccharide Nanoparticles for Gene Delivery
3.5 Practical Application of Gene Delivery Systems
3.6 Polysaccharide-Based Nanoparticles
3.6.1 Chitosan
3.6.2 Dendrimers
3.6.3 Alginate
3.6.4 Hyaluronan
3.6.5 Pullulan
3.6.6 Pectin
3.6.7 Heparin
3.6.8 Other Polysaccharides
3.7 DNA Delivery
3.7.1 siRNA Delivery
3.8 Conclusion
References
4. Hydrogel-Based Gene TherapyShweta Kumari, Dipti Jena, Vedant Kumar Prajapati, Shashi Ranjan Singh and Garima Tripathi
4.1 Introduction
4.2 Gene Therapy
4.2.1 Nonviral Vectors
4.2.2 Viral Vectors
4.3 In Vivo Gene Therapy Using Hydrogels
4.4 Encapsulating Cells in Hydrogels for Gene Therapy Delivery
4.5 Hydrogels for Integrative Tissue Engineering and Cell Delivery
4.6 Biocompatible Hydrogels for Transferring Cells
4.7 Using Hydrogels for Gene Therapy in Tissue Engineering-Based Drug
4.8 Human Gene Therapy that Uses Hydrogel as an Alternative Method of Delivering Genetic Material to Patients
4.9 Recent Advancement in Biocompatible Hydrogel
4.10 Applications of Hydrogel
4.11 Current Hydrogels in Clinical Trials
4.12 Conclusions
References
5. Progress and Prospects for Non-Viral Gene TherapyShashimala Tiwari
5.1 Introduction
5.2 Definition
5.3 Technology Overview for Non-Viral Gene Delivery
5.4 Chemical Carriers for Gene Transfer: Establishing Effective In Vivo Gene Delivery
5.5 Types of Gene Delivery
5.5.1 Lipid-Mediated Gene Delivery
5.5.2 Peptide-Mediated Gene Delivery
5.5.3 Polymer-Mediated Gene Transfer
5.6 Reduction of Immunological Responses Through Alteration of Delivery Method or DNA Structure
5.7 To Enable Long-Lasting Gene Expression, Self-Replicating, Tissue-Specific, and Integrating Plasmid Expression Systems are Designed
5.8 Hybrid Vector Systems to Improve Transfection and Lessen Cytotoxicity
5.8.1 Inorganic-Organic Hybrid Transmitters
5.8.2 Improved PEI Vectors
5.8.3 Peptide-Lipid Vectors or Inorganic-Lipid
5.8.4 Effects of In Vitro and In Vivo Performance on Characteristics of Gene Vectors and Formulation Parameters
5.9 Vehicle Material
5.9.1 The Proportion of Vehicle Components to Genes
5.9.2 Nanoparticle Size
5.10 Further Effects
5.11 Challenges and Prospects
5.12 Conclusion
References
6. Nanoparticles for Tumor Gene Therapy: Recent Advances and PerspectiveR. Shivhare, V. Sabale, A. Ingole and Neelam Jain
6.1 Introduction
6.2 Technologies for Gene Delivery
6.3 Cancer Treatment with Gene Therapy
6.4 Gene Therapy Using Nanotechnology
6.4.1 Polymeric Nanoparticles
6.4.2 Lipid Nanoparticles
6.4.3 Inorganic Nanoparticle
6.4.4 Gold Nanoparticles
6.4.5 Calcium Phosphate Nanoparticles
6.4.6 Iron Oxide Nanoparticles
6.4.7 Magnetic Nanoparticle
6.4.8 Zeolitic Imidazolate (ZI)
6.4.9 Silica Nanoparticles
6.4.10 Dendrimer
6.4.11 DOTAP-mPEG-PCL (DMP) Micelles
6.4.12 Stimulus Responsive Nanoparticles
6.4.13 Carbon Dots
6.4.14 Hybrid Nanoparticles
6.5 Challenges and Future Aspects
References
7. Effective Gene Transfer with Non-Viral VectorsAnil Kumar Mavi, Sonal Gaur, Neelesh Kumar, Avanish Kumar Shrivastav, Sankha Bhattacharya, Sateesh Belemkar, Saurabh Maru and Dhruv Kumar
7.1 Introduction
7.2 System Development for Delivering Genes
7.2.1 Virus-Based Vectors for Delivering Genes
7.2.2 Non-Viral Vector for Delivery Genes
7.3 Methods for Non-Viral Vector for Delivery of Genes
7.3.1 Physical Techniques for Delivering Non-Viral Genes
7.3.1.1 Microinjection
7.3.1.2 Needle Injection
7.3.1.3 Jet Gun
7.3.1.4 Gene Gun
7.3.1.5 Electroporation
7.3.1.6 Nucleofection
7.3.1.7 Sonoporation
7.3.1.8 Hydrodynamic Gene Transfer
7.3.1.9 Magnetoporation
7.3.1.10 Mechanical Massage
7.3.2 Chemical Methods for Delivering Non-Viral Genes
7.3.2.1 Polymer-Based Nano-Vectors
7.3.2.2 Protein-Based Vectors
7.3.2.3 Polysaccharide-Based Vectors
7.3.2.4 Liposomes
7.3.2.5 Inorganic Materials
7.4 Delivery System
7.4.1 Distribution Methods Using Non-Viral Nucleic Acids are Used for Localized Delivery
7.4.2 Polymeric Systems (Polyplex)
7.4.2.1 Lipid-Based Systems (Lipoplex)
7.4.2.2 Lipopolyplex
7.4.3 Delivery Methods for Nucleic Acids with Localized Release Naked Nucleic Acids Delivered Locally
7.4.3.1 Direct Method
7.4.3.2 Composite Method
7.4.3.3 Physical Methods
7.5 Current Methods for Nonviral Gene Delivery: Benefits and Drawbacks
7.6 Current Barriers for Non-Viral Vectors
7.7 Possibilities for Enhancing the Non-Viral Vector Delivery System
7.8 Conclusion
7.9 Future Relevance
References
8. Utilization of Chitosan for Gene DeliveryJohnson Olaleye Oladele
8.1 Introduction
8.2 Cationic Polymers-Based Gene Delivery Systems
8.2.1 Poly(L-lysine)-Based Gene Delivery Systems
8.2.2 Polysaccharides-Based Gene Delivery Systems
8.2.3 Poly(ethylenimine)s-Based Gene Delivery Systems
8.3 Chitosan and Its Derivatives in Gene Delivery Systems
8.3.1 Procedures of Preparation
8.3.2 Chitosan’s PEG Modification in Gene Delivery System
8.3.3 Derivatives of Chitosan in Gene Delivery
8.3.4 Factors that Affect Chitosan-Based Gene Delivery
8.4 Chitosan as Chemotherapeutic Drugs
8.5 Conclusion
References
9. Nanoparticles as Gene Vectors in Tumor TherapyEfstathia Triantafyllopoulou, Orestis Kontogiannis, Nefeli Lagopati, Natassa Pippa and Maria Gazouli
9.1 Introduction
9.2 Polymer-Based Nanocarriers: Their Technology and Recent Advances
9.2.1 Lipid-Based Nanocarriers: Their Technology and Recent Advances
9.2.2 Inorganic Nanocarriers: Their Technology and Recent Advances
9.3 Conclusions
References
10. Progress in Non-Viral Delivery of Nucleic Acid: Advancement in Biomedical TechnologyAnil Kumar Mavi, Manmohan Kumar, Amarjeet Singh, Mahendra Kumar Prajapati, Rakhi Khabiya, Saurabh Maru and Dhruv Kumar
10.1 Introduction
10.2 Physical Methods of Non-Viral Nucleic Acid Delivery System
10.2.1 Electroporation
10.2.2 Laserfection/Optical Transfection/Optotransfection
10.2.3 Gene Injection (or Biolistic Methods)
10.2.4 Magnetic Nanoparticles (MgNPs)
10.2.5 Sonoporation
10.2.6 Hydrodynamic Delivery
10.2.7 Microneedle
10.2.8 Microinjection
10.3 Advantages and Disadvantages of Physical Transfection
10.4 Chemical Methods of Non-Viral Nucleic Acid Delivery System
10.4.1 Inorganic Particles
10.4.2 Lipid-Based Nucleic Acid Delivery System
10.4.3 Polymer-Based Nucleic Acid Delivery System
10.4.4 Protein-Based Nucleic Acid Delivery System
10.4.5 Peptide-Based Nucleic Acid Delivery System
10.5 Advantages and Disadvantages of Chemical Transfection
10.6 Cellular Barriers for Nucleic Acid Delivery Faced by Non-Viral Vectors
10.7 Challenges and Limitations of Non-Viral Nucleic Acid Delivery System
10.8 Conclusion
References
11. The Junction of Biomaterials and Gene Therapy – Current Strategies and Future DirectionsRanjan Kumar Singh, Sunita Panchawat, Chennu M.M. Prasada Rao, Joohee Pradhan, Rajeswari Tanniru, Deepika Bairagee and Ajay Kumar Garg
11.1 Introduction
11.1.1 Gene Therapy
11.1.2 Ex Vivo
11.1.3 In Vivo Gene Therapy
11.2 Viral Gene Therapy
11.2.1 Retroviral Vectors
11.3 DNA Viral Vectors
11.3.1 Adenoviral Vectors
11.4 Adeno-Associated Viral Vectors
11.4.1 Herpes Simplex Viral Vectors
11.4.2 Other Types of Viral Vectors
11.5 Non-Viral Gene Therapy
11.5.1 Advances in Gene Delivery
11.6 Recent Advances in the Development of Gene Delivery Systems
11.7 Development of Gene Delivery Systems
11.7.1 Viral Vector Gene Delivery Systems
11.8 Viral Vectors Based on DNA for Gene Delivery Systems
11.9 Viral Vectors Based on RNA for Gene Delivery Systems
11.10 Oncolytic Viral Vectors for Gene Delivery Systems
11.10.1 Non-Viral Vector Gene Delivery System
11.10.2 Nano-Sized Organic-In-Organic Particles
11.10.2.1 Cerasomes
11.10.2.2 Magnetoliposomes
11.10.2.3 Nanomachine
11.11 Practical Application of Gene Delivery Methods
11.11.1 Gene Delivery Systems Based on Cationic Polymers
11.11.2 Gene Delivery Systems Based on Polysaccharides
11.11.3 Poly(Ethylenimine)-Based Gene Delivery
11.11.4 Polymer-Based Gene Delivery (L-lysine)
11.12 Conclusion
References
12. Utilization of Silk for Gene DeliverySwatantra K. S. Kushwaha, Shruti Khare and Neelottama Kushwaha
12.1 Introduction
12.2 Dimensional Structure of Silk
12.3 Properties of Silk
12.3.1 Biocompatibility
12.3.2 Mechanical Strength
12.3.3 Stability
12.3.4 Degradability
12.4 Extraction of Fibroin from Silk Worm
12.5 Fabrication of Silk in Different Therapeutics Carriers
12.5.1 Drug Delivery System Based on Hydrogels
12.5.2 Silk Nanoparticles
12.5.3 Silk Films
12.5.4 Silk Nanofibers
12.6 Utilization of Silk for Gene Therapy
12.7 Properties of Silk Fibroin as Biomaterial
12.7.1 Silk Fibroin’s Amino Acid Composition and Derivatization
12.7.2 Chain Length and Molecular Weight of Silk Fibroin
12.7.3 Solubility of Silk Fibroin
12.7.4 Hydrophobic and Crystal Nature of Silk Fibroin
12.7.5 Biocompatibility of Silk Fibroin
12.7.6 Thermal Stability of Silk Fibroin
12.8 Summary of Silk-Based Formulations for Gene Delivery
12.9 Examples of Some Delivery Approaches which Utilizes Silk as a Biomaterial for Gene Delivery
12.9.1 Bioengineered Silk and Elastin-Based Materials for Gene and Delivery Medication
12.9.2 Multifunctional Spider Silk Polymers for Gene Delivery
12.9.3 Insertable Silk Fabricated with Silk Fiber for Drug Delivery
12.9.4 Gene Carriers Based on Spider Silk that Target Specific Tumor Cells
12.9.5 Nuclear Targeting Using Bioengineered Silk Gene Delivery System
12.9.6 Layer-by-Layer Microcapsules Made of Silk Fibroin for Localized Gene Delivery
12.10 Some Highlights of Silk Fibroin
12.11 Conclusion
References
13. Challenges and Emerging Problems in Nanomedicine Mediated Gene TherapyShalini Bhatt, Neha Faridi, Rakshit Pathak, Vinay Deep Punetha and Mayank Punetha
13.1 Introduction
13.2 Why Nanomedicine Over Traditional Drugs?
13.3 Nanomedicine for Gene Therapy
13.3.1 Viral Vectors
13.3.2 Nanoparticle-Based Vectors
13.4 Complications in Nanomedicine-Mediated Gene Therapy
13.4.1 Problems in the Systemic Circulation
13.4.2 Problem in Selective Binding with Target Cell
13.4.3 Intracellular Barriers
13.5 Challenges in the Clinical Translation of Nanomedicines
13.5.1 Biological Challenges
13.5.2 Large-Scale Production
13.5.3 Immunological Response
13.5.4 Overcoming PEG Dilemma
13.5.5 Biocompatibility and Safety
13.5.6 Intellectual Property (IP)
13.5.7 Government Regulations
13.6 Conclusion
References
14. Biomaterials-Based Vaccination in Cancer TherapyRishav Sharma, Rishabha Malviya and Sonali Sundram
14.1 Introduction
14.2 Tumor-Associated Antigens
14.3 Vaccine Delivery
14.4 Dendritic Cells
14.5 In Vitro Generation of Dendritic Cells
14.6 Usage of RNA
14.7 RNA-Pulsed DCs as Vaccines
14.8 RNA Vaccines
14.9 Optimization of Immunotherapy
14.10 Cancer Treatment Through RNA Interference
14.11 Conclusion
References
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