Gene Editing for CEP290-Associated Retinal Degeneration

A study assessed the use of gene editing to treat CEP290 -associated inherited retinal degeneration, a disease that causes severe early-onset vision loss in childhood, and showed safety and early evidence of efficacy.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2024-06, Vol.390 (21), p.1972-1984
Hauptverfasser:	Pierce, Eric A., Aleman, Tomas S., Jayasundera, Kanishka T., Ashimatey, Bright S., Kim, Keunpyo, Rashid, Alia, Jaskolka, Michael C., Myers, Rene L., Lam, Byron L., Bailey, Steven T., Comander, Jason I., Lauer, Andreas K., Maguire, Albert M., Pennesi, Mark E.
Format:	Artikel
Sprache:	eng
Schlagworte:	Acuity Adverse events Age Childhood Diseases Children Consent CRISPR Drug dosages Eye FDA approval Gene Editing Genetics Genetics General Genome editing Hypothesis testing Mobility Ophthalmology Ophthalmology General Pediatrics Pediatrics General Photoreceptors Quality of life Questionnaires Retina Retinal degeneration Vision Visual acuity Visual impairment Visual perception
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Beschreibung
Zusammenfassung:	A study assessed the use of gene editing to treat CEP290 -associated inherited retinal degeneration, a disease that causes severe early-onset vision loss in childhood, and showed safety and early evidence of efficacy.
ISSN:	0028-4793 1533-4406 1533-4406
DOI:	10.1056/NEJMoa2309915