期刊
COLD SPRING HARBOR PERSPECTIVES IN MEDICINE
卷 5, 期 5, 页码 -出版社
COLD SPRING HARBOR LAB PRESS, PUBLICATIONS DEPT
DOI: 10.1101/cshperspect.a017160
关键词
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资金
- National Institutes of Health (NIH) Directors New Innovator Award [1-DP2-OD007483-01]
- NEI [EY017451, F32 EY022834]
- Howard Hughes Medical Institute
- Foundation Fighting Blindness
- Stephen A. Wynn Foundation
- Grousbeck Family Foundation
- Leo, Jacques & Marion Hauser Family Vision Restoration Fund
The ability to provide early clinical intervention for inherited disorders is heavily dependent on knowledge of a patient's disease-causing mutations and the resultant pathophysiologic mechanism(s). Without knowing a patient's disease-causing gene, and how gene mutations alter the health and functionality of affected cells, itwould be difficult to develop and deliver patient-specific molecular or small molecule therapies. Many believe that the field of stem cell biology holds the keys to the future development of disease-, patient-, and cell-specific therapies. In the case of the eye, which is susceptible to an extremely common late-onset degenerative disease known as age-related macular degeneration, stem cell-based therapies could increase the quality of life for millions of patientsworldwide. Furthermore, autologous, patient-specific induced pluripotent stem cells could be a viable source to treat rare Mendelian retinal degenerative diseases such as retinitis pigmentosa, Stargardt disease, and Best disease, to name a few.
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