Retinal ganglion cell gene therapy and visual system repair

M Hellstrom, AR Harvey - Current gene therapy, 2011 - ingentaconnect.com
Recent clinical trials have shown that the use of replication deficient viral vectors to
genetically modify cells in the retina can be of therapeutic benefit in the treatment of certain …

Molecular Signature of Astrocytes for Gene Delivery by the Synthetic Adeno‐Associated Viral Vector rAAV9P1

A Bauer, M Puglisi, D Nagl, JA Schick… - Advanced …, 2022 - Wiley Online Library
Astrocytes have crucial functions in the central nervous system (CNS) and are major players
in many CNS diseases. Research on astrocyte‐centered diseases requires efficient and well …

[HTML][HTML] Transduction optimization of AAV vectors for human gene therapy of glaucoma and their reversed cell entry characteristics

L Rodriguez-Estevez, P Asokan, T Borrás - Gene therapy, 2020 - nature.com
The trabecular meshwork (TM) of the eye is responsible for maintaining physiological
intraocular pressure (IOP). Dysfunction of this tissue results in elevated IOP, subsequent …

[HTML][HTML] Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia

X Dai, Y He, H Zhang, Y Zhang, Y Liu, M Wang… - PLoS …, 2017 - journals.plos.org
Adeno-associated virus (AAV) vectors are important gene delivery tools for the treatment of
many recessively inherited retinal diseases. For example, a wild-type (WT) AAV5 vector can …

Adeno-associated virus vectors efficiently transduce mouse and rabbit sensory neurons coinfected with herpes simplex virus 1 following peripheral inoculation

ZL Watson, MK Ertel, AS Lewin, SS Tuli… - Journal of …, 2016 - Am Soc Microbiol
Following infection of epithelial tissues, herpes simplex virus 1 (HSV-1) virions travel via
axonal transport to sensory ganglia and establish a lifelong latent infection within neurons …

Post-translational modification of heat-shock protein 90: impact on chaperone function

BT Scroggins, L Neckers - Expert opinion on drug discovery, 2007 - Taylor & Francis
Heat-shock protein 90 (Hsp90) is a molecular chaperone required for the stability and
function of many signaling proteins that are often activated, mutated or overexpressed in …

Tissue and cell-type-specific transduction using rAAV vectors in lung diseases

K Kochergin-Nikitsky, L Belova, A Lavrov… - Journal of molecular …, 2021 - Springer
Gene therapy of genetically determined diseases, including some pathologies of the
respiratory system, requires an efficient method for transgene delivery. Recombinant adeno …

[HTML][HTML] Development of novel AAV serotype 6 based vectors with selective tropism for human cancer cells

R Sayroo, D Nolasco, Z Yin, Y Colon-Cortes, M Pandya… - Gene Therapy, 2016 - nature.com
Viral vectors-based gene therapy is an attractive alternative to common anti-cancer
treatments. In the present studies, AAV serotype 6 vectors were identified to be particularly …

Development of an anti-angiogenic therapeutic model combining scAAV2-delivered siRNAs and noninvasive photoacoustic imaging of tumor vasculature …

Q Ruan, L Xi, SL Boye, S Han, ZJ Chen, WW Hauswirth… - Cancer letters, 2013 - Elsevier
We aimed to develop an anti-angiogenic model for breast cancer by combining (1) siRNA-
based therapy delivered by self-complementary adeno-associated virus serotype 2 …

Single-cell transcriptome analysis of mouse liver cell-specific tropism and transcriptional dysregulation following intravenous administration of AAVrh. 10 vectors

D Zhu, MR Rostami, W Zuo, PL Leopold… - Human gene …, 2020 - liebertpub.com
Capitalizing on liver tropism of adeno-associated viral (AAV) vectors, intravenous vector
administration is commonly used to genetically modify hepatocytes, a strategy currently in …