Let there be light: gene and cell therapy for blindness
Retinal degenerative diseases are a leading cause of irreversible blindness. Retinal cell
death is the main cause of vision loss in genetic disorders such as retinitis pigmentosa …
death is the main cause of vision loss in genetic disorders such as retinitis pigmentosa …
Cellular transduction mechanisms of adeno-associated viral vectors
GE Berry, A Asokan - Current opinion in virology, 2016 - Elsevier
Highlights•AAV exploits a spectrum of endocytic pathways after cellular uptake.•AAV
trafficking through the trans-Golgi network precedes nuclear entry.•Second-strand synthesis …
trafficking through the trans-Golgi network precedes nuclear entry.•Second-strand synthesis …
Controlling AAV tropism in the nervous system with natural and engineered capsids
MJ Castle, HT Turunen, LH Vandenberghe… - Gene Therapy for …, 2016 - Springer
More than one hundred naturally occurring variants of adeno-associated virus (AAV) have
been identified, and this library has been further expanded by an array of techniques for …
been identified, and this library has been further expanded by an array of techniques for …
Insight into the mechanisms of enhanced retinal transduction by the engineered AAV2 capsid variant‐7m8
H Khabou, M Desrosiers, C Winckler… - Biotechnology and …, 2016 - Wiley Online Library
Recently, we described a modified AAV2 vector—AAV2‐7m8—having a capsid‐displayed
peptide insertion of 10 amino acids with enhanced retinal transduction properties. The …
peptide insertion of 10 amino acids with enhanced retinal transduction properties. The …
Heparan sulfate binding promotes accumulation of intravitreally delivered adeno-associated viral vectors at the retina for enhanced transduction but weakly influences …
KT Woodard, KJ Liang, WC Bennett… - Journal of …, 2016 - Am Soc Microbiol
Many adeno-associated virus (AAV) serotypes efficiently transduce the retina when
delivered to the subretinal space but show limited success when delivered to the vitreous …
delivered to the subretinal space but show limited success when delivered to the vitreous …
AAV-mediated transduction and targeting of retinal bipolar cells with improved mGluR6 promoters in rodents and primates
Q Lu, TH Ganjawala, E Ivanova, JG Cheng, D Troilo… - Gene therapy, 2016 - nature.com
Adeno-associated virus (AAV) vectors have been a powerful gene delivery vehicle to the
retina for basic research and gene therapy. For many of these applications, achieving cell …
retina for basic research and gene therapy. For many of these applications, achieving cell …
Adeno-associated virus virions with variant capsid and methods of use thereof
DV Schaffer, RR Klimczak, JT Koerber… - US Patent …, 2016 - Google Patents
US9458517B2 - Adeno-associated virus virions with variant capsid and methods of use thereof
- Google Patents US9458517B2 - Adeno-associated virus virions with variant capsid and …
- Google Patents US9458517B2 - Adeno-associated virus virions with variant capsid and …
Safety and biodistribution evaluation in cynomolgus macaques of rAAV2tYF-PR1. 7-hCNGB3, a recombinant AAV vector for treatment of achromatopsia
G Ye, E Budzynski, P Sonnentag, TM Nork… - Human gene therapy …, 2016 - liebertpub.com
Applied Genetic Technologies Corporation (AGTC) is developing rAAV2tYF-PR1. 7-
hCNGB3, a recombinant adeno-associated viral (rAAV) vector expressing the human …
hCNGB3, a recombinant adeno-associated viral (rAAV) vector expressing the human …
Identification and validation of small molecules that enhance recombinant adeno-associated virus transduction following high-throughput screens
While the recent success of adeno-associated virus (AAV)-mediated gene therapy in clinical
trials is promising, challenges still face the widespread applicability of recombinant AAV …
trials is promising, challenges still face the widespread applicability of recombinant AAV …
Angiotensin-(1-7) decreases cell growth and angiogenesis of human nasopharyngeal carcinoma xenografts
N Pei, R Wan, X Chen, A Li, Y Zhang, J Li, H Du… - Molecular cancer …, 2016 - AACR
Abstract Angiotensin-(1-7)[Ang-(1-7)] is an endogenous, heptapeptide hormone acting
through the Mas receptor (MasR), with antiproliferative and antiangiogenic properties …
through the Mas receptor (MasR), with antiproliferative and antiangiogenic properties …