Gene therapy tools for brain diseases

S Ingusci, G Verlengia, M Soukupova… - Frontiers in …, 2019 - frontiersin.org
Neurological disorders affecting the central nervous system (CNS) are still incompletely
understood. Many of these disorders lack a cure and are seeking more specific and effective …

α-Synuclein and neuronal cell death

T Yasuda, Y Nakata, H Mochizuki - Molecular neurobiology, 2013 - Springer
Parkinson's disease (PD) is a progressive neurodegenerative disorder affecting∼ 1% of
people over the age of 65. Neuropathological hallmarks of PD are prominent loss of …

[图书][B] Ophthalmology

M Yanoff, JS Duker - 2009 - books.google.com
Based on valuable customer feedback, Doctors Yanoff and Duker have streamlined their
best-selling reference, Ophthalmology, to zero in on just the clinical answers you need in …

AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method

RL Klein, RD Dayton, JB Tatom, KM Henderson… - Molecular therapy, 2008 - cell.com
We compared adeno-associated virus (AAV) serotypes for expression levels of green
fluorescent protein (GFP) in the adult rat hippocampus by biophotonic imaging. Preparations …

Insulin‐like growth factor 2 reverses memory and synaptic deficits in APP transgenic mice

M Pascual‐Lucas, S Viana da Silva… - EMBO molecular …, 2014 - embopress.org
Insulin‐like growth factor 2 (IGF2) was recently found to play a critical role in memory
consolidation in rats and mice, and hippocampal or systemic administration of recombinant …

AAV's and uses thereof

G Gao, T Flotte, J Xie - US Patent 8,734,809, 2014 - Google Patents
The invention in some aspects relates to recombinant adeno-associated viruses having
distinct tissue targeting capabilities. In some aspects, the invention relates to gene transfer …

High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines

DM Markusic, RW Herzog, GV Aslanidi, BE Hoffman… - Molecular Therapy, 2010 - cell.com
Elimination of specific surface-exposed single tyrosine (Y) residues substantially improves
hepatic gene transfer with adeno-associated virus type 2 (AAV2) vectors. Here …

Immune responses to adenovirus and adeno-associated vectors used for gene therapy of brain diseases: the role of immunological synapses in understanding the …

PR Lowenstein, RJ Mandel, W Xiong… - Current gene …, 2007 - ingentaconnect.com
Researchers have conducted numerous pre-clinical and clinical gene transfer studies using
recombinant viral vectors derived from a wide range of pathogenic viruses such as …

Recombinant adeno-associated virus serotype 6 (rAAV2/6)-mediated gene transfer to nociceptive neurons through different routes of delivery

C Towne, M Pertin, AT Beggah, P Aebischer… - Molecular …, 2009 - journals.sagepub.com
Background Gene transfer to nociceptive neurons of the dorsal root ganglia (DRG) is a
promising approach to dissect mechanisms of pain in rodents and is a potential therapeutic …

Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6

C Towne, BL Schneider, D Kieran, DE Redmond… - Gene therapy, 2010 - nature.com
Retrograde transport of viral vectors in the rodent spinal cord provides a powerful means to
administer a therapeutic transgene from the innervated musculature. With the aim of scaling …