Detalhe da pesquisa
1.
Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain.
Gene Ther
; 25(3): 205-219, 2018 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-29785047
2.
Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial.
Lancet
; 390(10089): 50-61, 2017 07 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28526489
3.
CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10.
Mol Ther
; 25(2): 331-341, 2017 02 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28109959
4.
Soluble FLT1 Gene Therapy Alleviates Brain Arteriovenous Malformation Severity.
Stroke
; 48(5): 1420-1423, 2017 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-28325846
5.
Ocular gene therapy for neovascular AMD: a new era? - Authors' reply.
Lancet
; 390(10108): 2140, 2017 11 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-29143750
6.
AAV Gene Augmentation of Truncated Complement Factor H Differentially Rescues Ocular Complement Dysregulation in a Mouse Model.
Invest Ophthalmol Vis Sci
; 64(10): 25, 2023 07 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-37471073
7.
Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis.
Mol Ther Methods Clin Dev
; 28: 129-145, 2023 Mar 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-36654798
8.
Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule.
Mol Ther
; 19(2): 260-5, 2011 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-20978476
9.
Preclinical safety evaluation of AAV2-sFLT01- a gene therapy for age-related macular degeneration.
Mol Ther
; 19(2): 326-34, 2011 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-21119620
10.
Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases.
Nat Commun
; 12(1): 4219, 2021 07 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-34244505
11.
Probing the vibrational dynamics of high-lying electronic states using pump-degenerate four-wave mixing.
Phys Chem Chem Phys
; 12(6): 1351-6, 2010 Feb 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-20119613
12.
Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes.
Mol Ther Methods Clin Dev
; 9: 33-46, 2018 Jun 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-29349097
13.
Addendum to: Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases.
Nat Commun
; 14(1): 3503, 2023 06 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-37311765
14.
Development of a cellular model of rod opsin retinitis pigmentosa.
Adv Exp Med Biol
; 723: 573-9, 2012.
Artigo
em Inglês
| MEDLINE | ID: mdl-22183380
15.
AAVS1-Targeted Plasmid Integration in AAV Producer Cell Lines.
Hum Gene Ther Methods
; 28(3): 124-138, 2017 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-28504553
16.
BEST Disease and Gene Therapy.
Int Ophthalmol Clin
; 61(4): 167-172, 2021 10 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34584054
17.
Adenovirus-mediated expression of p35 prevents hypoxia/reoxygenation injury by reducing reactive oxygen species and caspase activity.
Cardiovasc Res
; 55(2): 309-19, 2002 Aug 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-12123770
18.
Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors.
Hum Gene Ther Methods
; 26(6): 228-42, 2015 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-26414997
19.
Myocardial expression of baculoviral p35 alleviates doxorubicin-induced cardiomyopathy in rats.
Hum Gene Ther
; 14(10): 947-57, 2003 Jul 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-12869213
20.
Gene Therapies for Neovascular Age-Related Macular Degeneration.
Cold Spring Harb Perspect Med
; 5(7): a017335, 2014 Dec 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-25524721