Viral vectors: from virology to transgene expression
D Bouard, N Alazard‐Dany… - British journal of …, 2009 - Wiley Online Library
In the late 1970s, it was predicted that gene therapy would be applied to humans within a
decade. However, despite some success, gene therapy has still not become a routine …
decade. However, despite some success, gene therapy has still not become a routine …
Immune responses to AAV in clinical trials
F Mingozzi, KA High - Current gene therapy, 2011 - ingentaconnect.com
Findings in the first clinical trial in which an adeno-associated virus (AAV) vector was
introduced into the liver of human subjects highlighted an issue not previously identified in …
introduced into the liver of human subjects highlighted an issue not previously identified in …
[HTML][HTML] Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular …
Y Lai, GD Thomas, Y Yue, HT Yang… - The Journal of …, 2009 - Am Soc Clin Investig
Sarcolemma-associated neuronal NOS (nNOS) plays a critical role in normal muscle
physiology. In Duchenne muscular dystrophy (DMD), the loss of sarcolemmal nNOS leads to …
physiology. In Duchenne muscular dystrophy (DMD), the loss of sarcolemmal nNOS leads to …
Production, purification and characterization of adeno-associated vectors
E Ayuso, F Mingozzi, F Bosch - Current gene therapy, 2010 - ingentaconnect.com
The use of recombinant adeno-associated viral vectors (rAAVs) as gene transfer tools has
increased dramatically during the past several years, establishing AAV as the vector of …
increased dramatically during the past several years, establishing AAV as the vector of …
[HTML][HTML] Immune responses to AAV-vectors, the Glybera example from bench to bedside
V Ferreira, H Petry, F Salmon - Frontiers in immunology, 2014 - frontiersin.org
Alipogene tiparvovec (Glybera®) is an adeno-associated virus serotype 1 (AAV1)-based
gene therapy that has been developed for the treatment of patients with lipoprotein lipase …
gene therapy that has been developed for the treatment of patients with lipoprotein lipase …
Systemic delivery of adeno-associated viral vectors
D Duan - Current opinion in virology, 2016 - Elsevier
Highlights•Only intravascular delivery can truly change the course of systemic
diseases.•AAV has the capacity to escape from the blood and lead to bodywide gene …
diseases.•AAV has the capacity to escape from the blood and lead to bodywide gene …
Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration
Adeno-associated virus (AAV) serotype-9 (AAV-9) has attracted great attention as an optimal
vehicle for body-wide gene delivery. Here we examined the effect of animal age (newborn …
vehicle for body-wide gene delivery. Here we examined the effect of animal age (newborn …
Immune responses to AAV in clinical trials
F Mingozzi, KA High - Current gene therapy, 2007 - ingentaconnect.com
Recent findings in a clinical trial in which an adeno-associated virus (AAV) vector
expressing coagulation factor IX (F. IX) was introduced into the liver of hemophilia B subjects …
expressing coagulation factor IX (F. IX) was introduced into the liver of hemophilia B subjects …
Can adeno-associated viral vectors deliver effectively large genes?
P Tornabene, I Trapani - Human gene therapy, 2020 - liebertpub.com
Gene therapy with adeno-associated viral (AAV) vectors has reached the clinical stage for
many inherited and acquired diseases. However, due to a cargo capacity limited to< 5 kb …
many inherited and acquired diseases. However, due to a cargo capacity limited to< 5 kb …
AAV. Dysferlin overlap vectors restore function in dysferlinopathy animal models
PC Sondergaard, DA Griffin, ER Pozsgai… - Annals of clinical …, 2015 - Wiley Online Library
Objective Dysferlinopathies are a family of untreatable muscle disorders caused by
mutations in the dysferlin gene. Lack of dysferlin protein results in progressive dystrophy …
mutations in the dysferlin gene. Lack of dysferlin protein results in progressive dystrophy …