scholarly journals Treatment of Leber Congenital Amaurosis Due toRPE65Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

2008 ◽  
Vol 19 (10) ◽  
pp. 979-990 ◽  
Author(s):  
William W. Hauswirth ◽  
Tomas S. Aleman ◽  
Shalesh Kaushal ◽  
Artur V. Cideciyan ◽  
Sharon B. Schwartz ◽  
...  
Keyword(s):  
Phase I ◽  
Phase I Trial ◽  
Gene Vector ◽  
Short Term ◽  
Adeno Associated Virus ◽  
Leber Congenital Amaurosis ◽  
Virus Gene ◽  
Subretinal Injection

Phase I Trial of Leber Congenital Amaurosis due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results

2008 ◽  
Vol 0 (ja) ◽  
pp. 081015093227032 ◽  
Author(s):  
William Hauswirth ◽  
Tomas S Aleman ◽  
Shalesh Kaushal ◽  
Artur V Cideciyan ◽  
Sharon B Schwartz ◽  
...  
Keyword(s):  
Phase I ◽  
Phase I Trial ◽  
Gene Vector ◽  
Short Term ◽  
Adeno Associated Virus ◽  
Leber Congenital Amaurosis ◽  
Virus Gene ◽  
Subretinal Injection

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

2016 ◽  
Vol 135 (3) ◽  
pp. 327-343 ◽  
Author(s):  
Nicola G. Ghazi ◽  
Emad B. Abboud ◽  
Sawsan R. Nowilaty ◽  
Hisham Alkuraya ◽  
Abdulrahman Alhommadi ◽  
...  
Keyword(s):  
Retinitis Pigmentosa ◽  
Phase I ◽  
Phase I Trial ◽  
Gene Vector ◽  
Adeno Associated Virus ◽  
Virus Gene ◽  
Subretinal Injection

Phase I Trial of Intramuscular Injection of a Recombinant Adeno-associated Virus Serotype 2 ?1-Antitrypsin (AAT) Vector in AAT-Deficient Adults

2006 ◽  
Vol 0 (0) ◽  
pp. 061121084323001
Author(s):  
Mark L. Brantly ◽  
L. Terry Spencer ◽  
Margaret Humphries ◽  
Thomas J. Conlon ◽  
Carolyn T. Spencer ◽  
...  
Keyword(s):  
Phase I ◽  
Intramuscular Injection ◽  
Phase I Trial ◽  
Adeno Associated Virus ◽  
Virus Serotype

scholarly journals Phase I trial of tremelimumab in combination with short-term androgen deprivation in patients with PSA-recurrent prostate cancer

2011 ◽  
Vol 61 (7) ◽  
pp. 1137-1147 ◽  
Author(s):  
Douglas G. McNeel ◽  
Heath A. Smith ◽  
Jens C. Eickhoff ◽  
Joshua M. Lang ◽  
Mary Jane Staab ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Phase I ◽  
Phase I Trial ◽  
Androgen Deprivation ◽  
Recurrent Prostate Cancer ◽  
Short Term

scholarly journals CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis

2019 ◽  
Vol 5 (10) ◽  
pp. eaax1210 ◽  
Author(s):  
Dong Hyun Jo ◽  
Dong Woo Song ◽  
Chang Sik Cho ◽  
Un Gi Kim ◽  
Kyu Jun Lee ◽  
...  
Keyword(s):  
Nonsense Mutation ◽  
Stop Codon ◽  
Retinal Pigment ◽  
Childhood Onset ◽  
Adeno Associated Virus ◽  
Leber Congenital Amaurosis ◽  
Homology Directed Repair ◽  
Disease Phenotypes ◽  
Recessive Mutations ◽  
Subretinal Injection

Leber congenital amaurosis (LCA), one of the leading causes of childhood-onset blindness, is caused by autosomal recessive mutations in several genes including RPE65. In this study, we performed CRISPR-Cas9–mediated therapeutic correction of a disease-associated nonsense mutation in Rpe65 in rd12 mice, a model of human LCA. Subretinal injection of adeno-associated virus carrying CRISPR-Cas9 and donor DNA resulted in >1% homology-directed repair and ~1.6% deletion of the pathogenic stop codon in Rpe65 in retinal pigment epithelial tissues of rd12 mice. The a- and b-waves of electroretinograms were recovered to levels up to 21.2 ± 4.1% and 39.8 ± 3.2% of their wild-type mice counterparts upon bright stimuli after dark adaptation 7 months after injection. There was no definite evidence of histologic perturbation or tumorigenesis during 7 months of observation. Collectively, we present the first therapeutic correction of an Rpe65 nonsense mutation using CRISPR-Cas9, providing new insight for developing therapeutics for LCA.

Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

The Lancet ◽  
2007 ◽  
Vol 369 (9579) ◽  
pp. 2097-2105 ◽  
Author(s):  
Michael G Kaplitt ◽  
Andrew Feigin ◽  
Chengke Tang ◽  
Helen L Fitzsimons ◽  
Paul Mattis ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Gene Therapy ◽  
Parkinson's Disease ◽  
Phase I ◽  
Phase I Trial ◽  
Open Label ◽  
Adeno Associated Virus ◽  
Open Label Phase

Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2α1-Antitrypsin (AAT) Vector in AAT-Deficient Adults

2006 ◽  
Vol 17 (12) ◽  
pp. 1177-1186 ◽  
Author(s):  
Mark L. Brantly ◽  
L. Terry Spencer ◽  
Margaret Humphries ◽  
Thomas J. Conlon ◽  
Carolyn T. Spencer ◽  
...  
Keyword(s):  
Phase I ◽  
Intramuscular Injection ◽  
Phase I Trial ◽  
Adeno Associated Virus ◽  
Virus Serotype

Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2?1-Antitrypsin (AAT) Vector in AAT-Deficient Adults

2006 ◽  
Vol 0 (0) ◽  
pp. 061211062938001
Author(s):  
Mark L. Brantly ◽  
L. Terry Spencer ◽  
Margaret Humphries ◽  
Thomas J. Conlon ◽  
Carolyn T. Spencer ◽  
...  
Keyword(s):  
Phase I ◽  
Intramuscular Injection ◽  
Phase I Trial ◽  
Adeno Associated Virus ◽  
Virus Serotype
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