Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial

Human Genetics

Volumn 135, Issue 3, 2016, Pages 327-343

  Ghazi, Nicola G ^a,h   Abboud, Emad B ^a   Nowilaty, Sawsan R ^a   Alkuraya, Hisham ^m   Alhommadi, Abdulrahman ^a   Cai, Huimin ^d,j,k   Hou, Rui ^j,k   Deng, Wen Tao ^e   Boye, Sanford L ^e   Almaghamsi, Abdulrahman ^a   Al Saikhan, Fahad ^a   Al Dhibi, Hassan ^a   Birch, David ⁿ   Chung, Christopher ^d   Colak, Dilek ^g   LaVail, Matthew M ^c   Vollrath, Douglas ^b   Erger, Kirsten ^f   Wang, Wenqiu ^j,k   Conlon, Thomas ^f   Zhang, Kang ^d,l   Hauswirth, William ^e   Alkuraya, Fowzan S ^g,i   more..

^a KING KHALED EYE SPECIALIST HOSPITAL   (Saudi Arabia)

^b STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e UNIVERSITY OF FLORIDA   (United States)

^f UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^g KING FAISAL SPECIALIST HOSPITAL AND RESEARCH CENTER   (Saudi Arabia)

^h UNIVERSITY OF VIRGINIA   (United States)

ⁱ ALFAISAL UNIVERSITY   (Saudi Arabia)

^j Chengdu JiaChuan Biomedicine Co   (China)

^k Guangzhou KangRui Biological Pharmaceutical Technology Company Ltd   (China)

^l VA SAN DIEGO HEALTHCARE SYSTEM   (United States)

^m AL IMAM MOHAMMAD IBN SAUD ISLAMIC UNIVERSITY IMSIU   (Saudi Arabia)

ⁿ RETINA FOUNDATION OF THE SOUTHWEST   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

MER PROTO ONCOGENE TYROSINE KINASE; PARVOVIRUS VECTOR; PROTEIN TYROSINE KINASE; UNCLASSIFIED DRUG; MERTK PROTEIN, HUMAN; ONCOPROTEIN;

ADOLESCENT; ADULT; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CLINICAL ARTICLE; CONTROLLED STUDY; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG SAFETY; EYE PHOTOGRAPHY; FEMALE; FULL FIELD STIMULUS THRESHOLD TEST; GENE; GENE MUTATION; GENE THERAPY; GENE TRANSFER; HAPLORHINI; HUMAN; INTRAOCULAR PRESSURE; KERATITIS; MALE; MERTK GENE; NONHUMAN; OPHTHALMOLOGY; PHASE 1 CLINICAL TRIAL; PRIORITY JOURNAL; RETINITIS PIGMENTOSA; SPECTRAL DOMAIN OPTICAL COHERENCE TOMOGRAPHY; SUBCAPSULAR CATARACT; SUBRETINAL DRUG ADMINISTRATION; VISUAL ACUITY; VISUAL THRESHOLD; ANIMAL; BIOASSAY; CLINICAL TRIAL; DEPENDOPARVOVIRUS; DISEASE MODEL; FOLLOW UP; GENE VECTOR; GENETICS; MACACA; METABOLISM; MIDDLE AGED; MUTATION; OPTICAL COHERENCE TOMOGRAPHY; POSTOPERATIVE COMPLICATIONS; PROCEDURES; SUBRETINAL FLUID; TREATMENT OUTCOME; YOUNG ADULT;

ADOLESCENT; ADULT; ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; ENDPOINT DETERMINATION; FEMALE; FOLLOW-UP STUDIES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MACACA; MALE; MIDDLE AGED; MUTATION; POSTOPERATIVE COMPLICATIONS; PROTO-ONCOGENE PROTEINS; RECEPTOR PROTEIN-TYROSINE KINASES; RETINITIS PIGMENTOSA; SUBRETINAL FLUID; TOMOGRAPHY, OPTICAL COHERENCE; TREATMENT OUTCOME; VISUAL ACUITY; YOUNG ADULT;

EID: 84958777137     PISSN: 03406717     EISSN: 14321203     Source Type: Journal    
DOI: 10.1007/s00439-016-1637-y     Document Type: Article

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