However, application of this technology has been limited by the high degree of unintended 'off-target' editing events. Manipulation of the human genome using CRISPR-Cas gene editors has been validated in early clinical trials to correct genetic diseases. This Small Business Innovation Research (SBIR) Phase II project will develop and validate a curative therapy for spinal muscular atrophy (SMA) using a novel and proprietary CRISPR-Cas precision gene editing system. This technology can be used to improve and even cure patients of genetic disease. ![]() The proposed technology will increase the safety and efficacy of gene editing by optimizing the precision of editing to correct the disease. ![]() The broader impact/commercial potential of this Small Business Innovation Research (SBIR) Phase II project will be the development of a curative therapy for spinal muscular atrophy using a novel and proprietary precision gene editing technology.
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