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CRISPR/Cas9 genome surgery for retinal diseases.
Xu, Christine L; Park, Karen Sophia; Tsang, Stephen H.
Afiliación
  • Xu CL; Edward S Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA; Jonas Children's Vision Care and the Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University, New York, NY, USA.
  • Park KS; Edward S Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA; Jonas Children's Vision Care and the Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University, New York, NY, USA.
  • Tsang SH; Edward S Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA; Jonas Children's Vision Care and the Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University, New York, NY, USA; Department of Pathology & Cell Biology, Institute of Human Nutrition, Columbia Stem Cell Initiative, College of Physicians and Surgeons, Columbia University, New York, NY, USA. Electronic address: sht2@cumc.columbia.edu.
Drug Discov Today Technol ; 28: 23-32, 2018 Aug.
Article en En | MEDLINE | ID: mdl-30205877
Retinal diseases that impair vision can impose heavy physical and emotional burdens on patients' lives. Currently, clustered regularly interspaced short palindromic repeats (CRISPR) is a prevalent gene-editing tool that can be harnessed to generate disease model organisms for specific retinal diseases, which are useful for elucidating pathophysiology and revealing important links between genetic mutations and phenotypic defects. These retinal disease models are fundamental for testing various therapies and are indispensible for potential future clinical trials. CRISPR-mediated procedures involving CRISPR-associated protein 9 (Cas9) may also be used to edit genome sequences and correct mutations. Thus, if used for future therapies, CRISPR/Cas9 genome surgery could eliminate the need for patients with retinal diseases to undergo repetitive procedures such as drug injections. In this review, we will provide an overview of CRISPR/Cas9, discuss the different types of Cas9, and compare Cas9 to other endonucleases. Furthermore, we will explore the many ways in which researchers are currently utilizing this versatile tool, as CRISPR/Cas9 may have far-reaching effects in the treatment of retinal diseases.
Asunto(s)

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Enfermedades de la Retina / Terapia Genética / Sistemas CRISPR-Cas / Edición Génica Límite: Humans Idioma: En Revista: Drug Discov Today Technol Año: 2018 Tipo del documento: Article País de afiliación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Enfermedades de la Retina / Terapia Genética / Sistemas CRISPR-Cas / Edición Génica Límite: Humans Idioma: En Revista: Drug Discov Today Technol Año: 2018 Tipo del documento: Article País de afiliación: Estados Unidos