| Chapter title |
Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders
|
|---|---|
| Chapter number | 30 |
| Book title |
Gene Therapy for Neurological Disorders
|
| Published in |
Methods in molecular biology, January 2016
|
| DOI | 10.1007/978-1-4939-3271-9_30 |
| Pubmed ID | |
| Book ISBNs |
978-1-4939-3270-2, 978-1-4939-3271-9
|
| Authors |
Gessler, Dominic J, Gao, Guangping, Dominic J. Gessler, Guangping Gao, Gessler, Dominic J. |
| Abstract |
Metabolic disorders comprise a large group of heterogeneous diseases ranging from very prevalent diseases such as diabetes mellitus to rare genetic disorders like Canavan Disease. Whether either of these diseases is amendable by gene therapy depends to a large degree on the knowledge of their pathomechanism, availability of the therapeutic gene, vector selection, and availability of suitable animal models. In this book chapter, we review three metabolic disorders of the central nervous system (CNS; Canavan Disease, Niemann-Pick disease and Phenylketonuria) to give examples for primary and secondary metabolic disorders of the brain and the attempts that have been made to use adeno-associated virus (AAV) based gene therapy for treatment. Finally, we highlight commonalities and obstacles in the development of gene therapy for metabolic disorders of the CNS exemplified by those three diseases. |
X Demographics
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United States | 2 | 100% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Science communicators (journalists, bloggers, editors) | 1 | 50% |
| Practitioners (doctors, other healthcare professionals) | 1 | 50% |
Mendeley readers
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 49 | 100% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Bachelor | 8 | 16% |
| Researcher | 7 | 14% |
| Student > Ph. D. Student | 7 | 14% |
| Other | 3 | 6% |
| Student > Master | 3 | 6% |
| Other | 7 | 14% |
| Unknown | 14 | 29% |
| Readers by discipline | Count | As % |
|---|---|---|
| Biochemistry, Genetics and Molecular Biology | 10 | 20% |
| Medicine and Dentistry | 7 | 14% |
| Neuroscience | 5 | 10% |
| Agricultural and Biological Sciences | 4 | 8% |
| Pharmacology, Toxicology and Pharmaceutical Science | 3 | 6% |
| Other | 4 | 8% |
| Unknown | 16 | 33% |