Title |
Emerging Gene Therapies for Genetic Hearing Loss
|
---|---|
Published in |
Journal of the Association for Research in Otolaryngology, August 2017
|
DOI | 10.1007/s10162-017-0634-8 |
Pubmed ID | |
Authors |
Hena Ahmed, Olga Shubina-Oleinik, Jeffrey R. Holt |
Abstract |
Gene therapy, or the treatment of human disease using genetic material, for inner ear dysfunction is coming of age. Recent progress in developing gene therapy treatments for genetic hearing loss has demonstrated tantalizing proof-of-principle in animal models. While successful translation of this progress into treatments for humans awaits, there is growing interest from patients, scientists, clinicians, and industry. Nonetheless, it is clear that a number of hurdles remain, and expectations for total restoration of auditory function should remain tempered until these challenges have been overcome. Here, we review progress, prospects, and challenges for gene therapy in the inner ear. We focus on technical aspects, including routes of gene delivery to the inner ear, choice of vectors, promoters, inner ear targets, therapeutic strategies, preliminary success stories, and points to consider for translating of these successes to the clinic. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 2 | 100% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Scientists | 1 | 50% |
Practitioners (doctors, other healthcare professionals) | 1 | 50% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 127 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Ph. D. Student | 17 | 13% |
Student > Bachelor | 17 | 13% |
Researcher | 15 | 12% |
Student > Master | 10 | 8% |
Unspecified | 10 | 8% |
Other | 30 | 24% |
Unknown | 28 | 22% |
Readers by discipline | Count | As % |
---|---|---|
Biochemistry, Genetics and Molecular Biology | 24 | 19% |
Medicine and Dentistry | 20 | 16% |
Neuroscience | 12 | 9% |
Agricultural and Biological Sciences | 10 | 8% |
Unspecified | 10 | 8% |
Other | 13 | 10% |
Unknown | 38 | 30% |