Correction of monogenic and common retinal disorders with gene therapy

Genes

Volumn 8, Issue 2, 2017, Pages

  Sengillo, Jesse D ^a,b,c   Justus, Sally ^a,b   Cabral, Thiago ^a,b,d,e   Tsang, Stephen H ^a,b,f  

^a New York Presbyterian Morgan Stanley Children's Hospital   (United States)

^b NEW YORK PRESBYTERIAN HOSPITAL   (United States)

^c SUNY DOWNSTATE MEDICAL CENTER   (United States)

^d FEDERAL UNIVERSITY OF ESPÍRITO SANTO   (Brazil)

^e FEDERAL UNIVERSITY OF SÃO PAULO   (Brazil)

^f Department of Neurology and the Taub Institute for Research on Alzheimer's Disease and the Aging Brain   (United States)

Author keywords

CRISPR; Gene therapy; Genome engineering; Inherited retinal disease; Retinal degeneration

Indexed keywords

GENE THERAPY AGENT;

AGE RELATED MACULAR DEGENERATION; CHOROIDEREMIA; CLINICAL TRIAL (TOPIC); COLOR BLINDNESS; CRISPR CAS SYSTEM; DISEASE CLASSIFICATION; GENE THERAPY; GENE VECTOR; GENETIC ENGINEERING; GENOME; HUMAN; LEBER CONGENITAL AMAUROSIS; LEBER HEREDITARY OPTIC NEUROPATHY; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; RETINA DISEASE; RETINITIS PIGMENTOSA; RETINOSCHISIS; REVIEW; STARGARDT DISEASE; SUBRETINAL DRUG ADMINISTRATION; USHER SYNDROME; VIRAL GENE DELIVERY SYSTEM; WET MACULAR DEGENERATION; X CHROMOSOME LINKED DISORDER;

EID: 85014704683     PISSN: None     EISSN: 20734425     Source Type: Journal    
DOI: 10.3390/genes8020053     Document Type: Review

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