Gene Therapy For The Central And Peripheral Nervous System

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Gene Therapy for the Central and Peripheral Nervous System

Author : Andrew P. Tosolini
Publisher : Frontiers Media SA
ISBN 13 : 2889454754
Total Pages : 220 pages
Book Rating : 4.8/5 (894 download)

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Book Synopsis Gene Therapy for the Central and Peripheral Nervous System by : Andrew P. Tosolini

Download or read book Gene Therapy for the Central and Peripheral Nervous System written by Andrew P. Tosolini and published by Frontiers Media SA. This book was released on 2018-05-10 with total page 220 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene therapy is at the forefront of current techniques that aim to re-establish functional connectivity, after an insult to the brain, spinal cord or peripheral nerves. Gene therapy makes the most of the existing cellular machinery and anatomical networks to facilitate molecular changes in DNA, RNA and proteins aiming to repair these disrupted connections. For instance, gene therapy is currently being used to target genes in conditions including spinal cord injury, amyotrophic lateral sclerosis, spinal muscular atrophy, stroke and multiple sclerosis, amongst others. The various delivery routes include viral-vectors, genetically modified cellular implants, naked DNA/RNA, liposomes, Cre-Lox recombination, optogenetics and nanoparticles. In particular, gene therapy aims to restore function by augmenting the expression of neuroprotective/axonal growth-promoting neurotrophic factors (e.g., BDNF, CNTF, NGF and GDNF, etc.). Furthermore, the downstream intracellular signalling pathways after receptor activation can also be targeted (e.g., mTor, MAPK, etc.). On the other hand, gene therapy can also be used to downregulate and/or remove faulty mutated genes, such as those contributing to disease progression or that inhibit axonal regeneration (e.g., SOD-1, TDP-43, Nogo-A, MAG, OmGP, etc.). Depending on the methodology, these genes, for instance, can be silenced, removed or replaced to alleviate the underlying pathology. As such, gene therapy can transform a largely toxic and inhibitory milieu surrounding a neuronal/axonal insult into a growth-permissive environment that will ultimately aid neuronal survival and functional regeneration. Moreover, gene therapy has the capacity to target non-neuronal cells and can be even used for neuroanatomical tract tracing. Ultimately, the principal outcome of gene therapy is to functionally restore damaged neuronal and/or axonal connections irrespective of the system it is being introduced in to. This Research Topic is devoted to work using gene therapy for the both the central and/or peripheral nervous system.

Gene Therapy of the Central Nervous System: From Bench to Bedside

Author : Michael G. Kaplitt
Publisher : Gulf Professional Publishing
ISBN 13 : 9780123976321
Total Pages : 380 pages
Book Rating : 4.9/5 (763 download)

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Book Synopsis Gene Therapy of the Central Nervous System: From Bench to Bedside by : Michael G. Kaplitt

Download or read book Gene Therapy of the Central Nervous System: From Bench to Bedside written by Michael G. Kaplitt and published by Gulf Professional Publishing. This book was released on 2006 with total page 380 pages. Available in PDF, EPUB and Kindle. Book excerpt: Few areas of biomedical research provide greater opportunities to capitalize upon the revolution in genomics and molecular biology than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy in the Brain: From Bench to Bedside represents the definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped to create the field as well as those who are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects which would be of interest to anyone who is currently using or contemplating exploring gene therapy for neurobiological applications. Basic gene transfer technologies are discussed, with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells. Numerous research applications are reviewed, particularly in complex fields such as behavioral neurobiology. Several preclinical areas are also covered which are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis. Among the most exciting advances in recent years has been the use of neurological gene therapy in human clinical trials, including Parkinson's disease, Canavan disease and Batten disease. Finally, readers will find "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use. This work provides up-to-date information and key insights into those gene therapy issues which are important to both scientists and clinicians focusing upon the brain and central nervous system.

Advancing Gene-Targeted Therapies for Central Nervous System Disorders

Author : National Academies of Sciences, Engineering, and Medicine
Publisher : National Academies Press
ISBN 13 : 0309495849
Total Pages : 93 pages
Book Rating : 4.3/5 (94 download)

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Book Synopsis Advancing Gene-Targeted Therapies for Central Nervous System Disorders by : National Academies of Sciences, Engineering, and Medicine

Download or read book Advancing Gene-Targeted Therapies for Central Nervous System Disorders written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2019-12-01 with total page 93 pages. Available in PDF, EPUB and Kindle. Book excerpt: On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.

Gene Therapy in the CNS – Progress and Prospects for Novel Therapies

Author : Casper René Gøtzsche
Publisher : Frontiers Media SA
ISBN 13 : 2889718522
Total Pages : 188 pages
Book Rating : 4.8/5 (897 download)

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Book Synopsis Gene Therapy in the CNS – Progress and Prospects for Novel Therapies by : Casper René Gøtzsche

Download or read book Gene Therapy in the CNS – Progress and Prospects for Novel Therapies written by Casper René Gøtzsche and published by Frontiers Media SA. This book was released on 2021-12-21 with total page 188 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Gene Transfer and Therapy in the Nervous System

Author : Fred H. Gage
Publisher : Springer Science & Business Media
ISBN 13 : 3642848427
Total Pages : 226 pages
Book Rating : 4.6/5 (428 download)

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Book Synopsis Gene Transfer and Therapy in the Nervous System by : Fred H. Gage

Download or read book Gene Transfer and Therapy in the Nervous System written by Fred H. Gage and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 226 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene transfer technology is a powerful tool for increasing our understandingof brain functions. It is also the basis of gene therapy, which is now technically possible for the correction of many human diseases, including several disorders of the nervous (and muscular) system such as Alzheimer's disease, Parkinson's disease, and dystrophy. This volume,which contains the proceedings of a symposium of the Fondation Ipsen, provides a unique view of the state of the art on different transgenes, vectors, target cells, and clinical applications related to the nervous system.

Gene Therapy for Neurological Disorders and Brain Tumors

Author : E. Antonio Chiocca
Publisher : Springer Science & Business Media
ISBN 13 : 1592594786
Total Pages : 458 pages
Book Rating : 4.5/5 (925 download)

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Book Synopsis Gene Therapy for Neurological Disorders and Brain Tumors by : E. Antonio Chiocca

Download or read book Gene Therapy for Neurological Disorders and Brain Tumors written by E. Antonio Chiocca and published by Springer Science & Business Media. This book was released on 1997-10-23 with total page 458 pages. Available in PDF, EPUB and Kindle. Book excerpt: Leading gene therapy researchers and clinicians illuminate the field-from basic vector technology to current and future clinical applications in neurology. The authoritative contributors provide cutting-edge reviews of the vectors available for gene transfer to the central nervous system, the strategies against CNS tumors, the potential strategies against neurologic disorder, and the limitations of today's gene therapy approaches. Also discussed are significant applications of gene therapy to brain tumors, Parkinson's disease, ischemia, and Huntington's chorea. Readers will learn the current delivery methods for transgenes, will learn the characteristics of transgene delivery vectors, and come to understand the therapy for both neuro-oncologic and neurologic disorders.

Genetic Manipulation of the Nervous System

Author :
Publisher : Elsevier
ISBN 13 : 9780080532400
Total Pages : 276 pages
Book Rating : 4.5/5 (324 download)

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Book Synopsis Genetic Manipulation of the Nervous System by :

Download or read book Genetic Manipulation of the Nervous System written by and published by Elsevier. This book was released on 1995-11-24 with total page 276 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neuroscience Perspectives provides multidisciplinary reviews of topics in one of the most diverse and rapidly advancing fields in the life sciences. Whether you are a new recruit to neuroscience, or an established expert, look to this series for 'one-stop' sources of the historical, physiological, pharmacological, biochemical, molecular biological and therapeutic aspects of chosen research areas. The recent development of Gene Therapy procedures which allow specific genes to be delivered to human patients who lack functional copies of them is of major therapeutic importance. In addition such gene delivery methods can be used in other organisms to define the function of particular genes. These studies are of particular interest in the nervous system where there are many incurable diseases like Alzheimer's and Parkinson's diseases which may benefit from therapies of this kind. Unfortunately gene delivery methods for use in the nervous system have lagged behind those in other systems due to the fact that the methods developed in other systems are often not applicable to cells like neurons which do not divide. This book discusses a wide range of methods which have now been developed to overcome these problems and allow safe and efficient delivery of particular genes to the brain. Methods discussed include virological methods, physical methods (such as liposomes) and the transplantation of genetically modified cells. In a single volume therefore this book provides a complete view of these methods and indicates how they can be applied to the development of therapies for treating previously incurable neurological disorders.

Pediatric Neurology Part III

Author : Patrick Aubourg
Publisher : Elsevier Inc. Chapters
ISBN 13 : 012808457X
Total Pages : 732 pages
Book Rating : 4.1/5 (28 download)

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Book Synopsis Pediatric Neurology Part III by : Patrick Aubourg

Download or read book Pediatric Neurology Part III written by Patrick Aubourg and published by Elsevier Inc. Chapters. This book was released on 2013-04-24 with total page 732 pages. Available in PDF, EPUB and Kindle. Book excerpt: Though heavily hyped in the early 1990s as the answer to many genetic diseases, gene therapy has largely failed to live up to its promises up to now. The scientific challenges of conveying replacement genes into the brain have prevented it from having an impact on more than a handful of conditions. In addition, gene therapy has been beset by safety problems. During the last 5 years, new vectors and techniques have started to resolve not only safety issues that once held gene therapy back but also markedly to improve efficient gene delivery within the brain and even the spinal cord. Brain gene therapy will not be suitable for every neurological disease, but considerable progress made in the field means we can now envisage that CNS gene therapy will be able to treat many neurological conditions that are not obviously treatable in any other way.

Axonal Regeneration in the Central Nervous System

Author : Nicholas A Ingoglia
Publisher : CRC Press
ISBN 13 : 9780824704100
Total Pages : 736 pages
Book Rating : 4.7/5 (41 download)

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Book Synopsis Axonal Regeneration in the Central Nervous System by : Nicholas A Ingoglia

Download or read book Axonal Regeneration in the Central Nervous System written by Nicholas A Ingoglia and published by CRC Press. This book was released on 2000-09-29 with total page 736 pages. Available in PDF, EPUB and Kindle. Book excerpt: Summarizing a review of research into factors that regulate, stimulate, and prevent regeneration in the central nervous system (CNS), this comprehensive reference progresses further into answering and resolving neuron capacity for axon regeneration in the mammalian CNS. Axonal Regeneration in the Central Nervous System analyzes axonal regeneration, reinnervation, and functional recovery in lower vertebrates examines the correlation between developmental age and the ability to regenerate considers mammalian neuron responses at the cell body, site of injury, and in the distal nerve, including apoptic cell death, and inflammatory and glial responses to injury reviews genomic responses to axotomy with a comparative description of transcribed genes from successfully regenerating neurons and neurons incapable of regrowth discusses how growing axons may induce the expression of genes in glia/Schwann cells following axotomy and regeneration describes the use of gene therapy to deliver trophic and survival factors to injured neurons explores the hospitable environments of the peripheral nerve, olfactory ensheathing cells, and fetal cell transplants for regeneration discusses results from applications of fetal CNS tissue to human spinal cord injuries and much more!

CNS Regeneration

Author : Mark H. Tuszynski
Publisher : Elsevier
ISBN 13 : 9780080529219
Total Pages : 678 pages
Book Rating : 4.5/5 (292 download)

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Book Synopsis CNS Regeneration by : Mark H. Tuszynski

Download or read book CNS Regeneration written by Mark H. Tuszynski and published by Elsevier. This book was released on 1998-10-27 with total page 678 pages. Available in PDF, EPUB and Kindle. Book excerpt: CNS Regeneration focuses on some of the leading current neurological disease models and methods for promoting central nervous system regeneration. Editors and authors are experts in the field, with experience in basic as well as applied neuroscience. In a comprehensive, logical manner, the book unites important basic science advances in neuroscience with novel medical strategies. The first comprehensive, authoritative volume on the topic of CNS regeneration Reviews current therapeutic approaches Editors and authors are experts in the field Appeals to those interested in basic science as well as those concerned with its medical application

Cancer of the Nervous System

Author : Peter McL. Black
Publisher : Lippincott Williams & Wilkins
ISBN 13 : 0781737311
Total Pages : 930 pages
Book Rating : 4.7/5 (817 download)

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Book Synopsis Cancer of the Nervous System by : Peter McL. Black

Download or read book Cancer of the Nervous System written by Peter McL. Black and published by Lippincott Williams & Wilkins. This book was released on 2005 with total page 930 pages. Available in PDF, EPUB and Kindle. Book excerpt: Thoroughly revised to reflect the latest advances in neurosurgery, radiation oncology, chemotherapy, biological therapy, and the basic sciences, the Second Edition of this highly acclaimed volume is the most comprehensive, current reference on tumors of the central and peripheral nervous system. More than 100 of the foremost authorities present multimodality treatment strategies for specific tumor types and examine the mechanisms of tumorigenesis. Coverage includes state-of-the-art information on image-guided surgery, local delivery systems, intraoperative imaging, proton beam therapy, conformal systems, radiosurgery, new drugs and biological agents, and cell cycle deregulation and chromosomal abnormalities in tumorigenesis. This edition contains over 400 illustrations.

Lentiviral Vectors

Author : Didier Trono
Publisher : Springer Science & Business Media
ISBN 13 : 9783540421900
Total Pages : 276 pages
Book Rating : 4.4/5 (219 download)

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Book Synopsis Lentiviral Vectors by : Didier Trono

Download or read book Lentiviral Vectors written by Didier Trono and published by Springer Science & Business Media. This book was released on 2001-12-14 with total page 276 pages. Available in PDF, EPUB and Kindle. Book excerpt: For the first time a compilation of chapters that depict the biological bases underlying the development of lentiviral vectors, the techniques involved in the manufacture of this new gene delivery tool, and its most promising applications.

Handbook of Innovations in Central Nervous System Regenerative Medicine

Author : António Salgado
Publisher : Elsevier
ISBN 13 : 0128180854
Total Pages : 712 pages
Book Rating : 4.1/5 (281 download)

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Book Synopsis Handbook of Innovations in Central Nervous System Regenerative Medicine by : António Salgado

Download or read book Handbook of Innovations in Central Nervous System Regenerative Medicine written by António Salgado and published by Elsevier. This book was released on 2020-06-10 with total page 712 pages. Available in PDF, EPUB and Kindle. Book excerpt: Handbook of Innovations in CNS Regenerative Medicine provides a comprehensive overview of the CNS regenerative medicine field. The book describes the basic biology and anatomy of the CNS and how injury and disease affect its balance and the limitations of the present therapies used in the clinics. It also introduces recent trends in different fields of CNS regenerative medicine, including cell transplantation, bio and neuro-engineering, molecular/pharmacotherapy therapies and enabling technologies. Finally, the book presents successful cases of translation of basic research to first-in-human trials and the steps needed to follow this path. Areas such as cell transplantation approaches, bio and neuro-engineering, molecular/pharmacotherapy therapies and enabling technologies are key in regenerative medicine are covered in the book, along with regulatory and ethical issues. Describes the basic biology and anatomy of the CNS and how injury and disease affect its balance Discusses the limitations of present therapies used in the clinics Introduces the recent trends in different fields of CNS regenerative medicine, including cell transplantation, bio and neuro-engineering, molecular/pharmacotherapy therapies, and enabling technologies Presents successful cases of translation of basic research to first-in-human trials, along with the steps needed to follow this path

Gene Therapy - Principles and Challenges

Author :
Publisher :
ISBN 13 : 9789535172680
Total Pages : pages
Book Rating : 4.1/5 (726 download)

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Book Synopsis Gene Therapy - Principles and Challenges by :

Download or read book Gene Therapy - Principles and Challenges written by and published by . This book was released on 2015 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:

Immunopharmacology

Author : Manzoor M. Khan
Publisher : Springer Science & Business Media
ISBN 13 : 0387779760
Total Pages : 275 pages
Book Rating : 4.3/5 (877 download)

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Book Synopsis Immunopharmacology by : Manzoor M. Khan

Download or read book Immunopharmacology written by Manzoor M. Khan and published by Springer Science & Business Media. This book was released on 2008-12-19 with total page 275 pages. Available in PDF, EPUB and Kindle. Book excerpt: During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.

A Guide to Human Gene Therapy

Author : Roland W. Herzog
Publisher : World Scientific
ISBN 13 : 9814280917
Total Pages : 415 pages
Book Rating : 4.8/5 (142 download)

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Book Synopsis A Guide to Human Gene Therapy by : Roland W. Herzog

Download or read book A Guide to Human Gene Therapy written by Roland W. Herzog and published by World Scientific. This book was released on 2010 with total page 415 pages. Available in PDF, EPUB and Kindle. Book excerpt: 1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne

Indwelling Neural Implants

Author : William M. Reichert
Publisher : CRC Press
ISBN 13 : 9781420009309
Total Pages : 296 pages
Book Rating : 4.0/5 (93 download)

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Book Synopsis Indwelling Neural Implants by : William M. Reichert

Download or read book Indwelling Neural Implants written by William M. Reichert and published by CRC Press. This book was released on 2007-12-17 with total page 296 pages. Available in PDF, EPUB and Kindle. Book excerpt: Despite enormous advances made in the development of external effector prosthetics over the last quarter century, significant questions remain, especially those concerning signal degradation that occurs with chronically implanted neuroelectrodes. Offering contributions from pioneering researchers in neuroprosthetics and tissue repair, Indwelling Neural Implants: Strategies for Contending with the In Vivo Environment examines many of these challenges, paying particular attention to how the healing of tissues surrounding an implant can impact the intended use of a device. The contributions are divided into four sections · Part one examines wound healing from the initial insertion trauma through the inflammatory and repair process, explaining how the action of healing varies throughout different areas of the body. · Part two considers various performance issues specific to particular implant components, including those that arise from the chemical, mechanical, thermal, and electrical impact on surrounding tissues. It discusses challenges that result from chronic tissue stimulation and heat effects that occur with on-chip and telemetric processing. · Part three presents both in vitro and in vivo approaches to assessing wound healing response to materials. It includes the contribution of the developer of a chronic hollow fiber membrane implant who explains how an in vivo model is used to assess molecular transport in brain tissue surrounding the implant. · The final section evaluates molecular and materials strategies for intervening in CNS wound repair and enhancing the electrical communication between the electrode surface and the surrounding tissue. It also presents novel approaches to nerve regeneration and repair. This seminal work provides researchers with an up-to-date account of the progress in the field that they can build upon to bring us closer to realizing the full value of neural implants in combating otherwise intractable human health problems.