Molecular Therapy: Methods & Clinical Development, 2021 Spinal muscular atrophy is a progressive, recessively inherited monogenic neurologic disease, the genetic root cause of which is the absence of a functional survival motor neuron 1 gene. Onasemnogene abeparvovec (formerly AVXS-101) is an adeno-associated
John W. Day +7 more
doaj +1 more source
Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus. [PDF]
, 2013 Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified before the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness.
Boye, S +8 more
core +1 more source
Neuroscience Insights, 2019 Recombinant adeno-associated virus (rAAV) vectors have emerged as the safe vehicles of choice for long-term gene transfer in mammalian nervous system. Recombinant adeno-associated virus–mediated localized gene transfer in adult nervous system following ...
Asad Jan +4 more
doaj +1 more source
Status and Prospect of Gene Therapy for Hemophilia
罕见病研究, 2022 Hemophilia is a congenital hemorrhagic disease caused by genetic abnormalities in coagulation factor Ⅷ or factor Ⅸ. Current conventional therapy to prevent bleeding requires frequent intravenous injections of coagulation factor concentrates from early ...
DAI Xinyue, ZHANG Lei
doaj +1 more source
Profiling of Glycan Receptors for Minute Virus of Mice in Permissive Cell Lines Towards Understanding the Mechanism of Cell Recognition [PDF]
, 2013 The recognition of sialic acids by two strains of minute virus of mice (MVM), MVMp (prototype) and MVMi (immunosuppressive), is an essential requirement for successful infection.
Agbandje-McKenna, M +12 more
core +1 more source
In vivo genetic manipulation of inner ear connexin expression by bovine adeno-Associated viral vectors [PDF]
, 2017 We have previously shown that in vitro transduction with bovine adeno-associated viral (BAAV) vectors restores connexin expression and rescues gap junction coupling in cochlear organotypic cultures from connexin-deficient mice that are models DFNB1 ...
Campioni, Matteo +7 more
core +1 more source
LDLR-Gene therapy for familial hypercholesterolaemia: Problems, progress, and perspectives [PDF]
, 2010 Coronary artery diseases (CAD) inflict a heavy economical and social burden on most populations and contribute significantly to their morbidity and mortality rates.
Al-Allaf, FA +5 more
core +3 more sources
The natural dietary genistein boosts bacteriophage-mediated cancer cell killing by improving phage-targeted tumor cell transduction [PDF]
, 2016 Gene therapy has long been regarded as a promising treatment for cancer. However, cancer gene therapy is still facing the challenge of targeting gene delivery vectors specifically to tumors when administered via clinically acceptable non-invasive ...
Albahrani, M +5 more
core +2 more sources
Intrabody Gene Therapy Ameliorates Motor, Cognitive, and Neuropathological Symptoms in Multiple Mouse Models of Huntington's Disease [PDF]
, 2009 Huntington's disease (HD) is an autosomal dominant neurodegenerative disease resulting from the expansion of a glutamine repeat in the huntingtin (Htt) protein.
Ko, Jan +2 more
core +2 more sources
Chapparvoviruses occur in at least three vertebrate classes and have a broad biogeographic distribution [PDF]
, 2017 Chapparvoviruses are a highly divergent group of parvoviruses (family Parvoviridae) that have recently been identified via metagenomic sampling of animal faeces.
Araujo, Jansen de +9 more
core +2 more sources