Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription
Scientific Reports, 2022 Duchenne muscular dystrophy is a severe neuromuscular disease causing a progressive muscle wasting due to mutations in the DMD gene that lead to the absence of dystrophin protein.
Amédée Mollard +11 more
doaj +1 more source
Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. [PDF]
, 2003 A systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice ...
Davidoff, AM +4 more
core +1 more source
Fluorescence Microscopy in Adeno-Associated Virus Research
Viruses, 2023 Research on adeno-associated virus (AAV) and its recombinant vectors as well as on fluorescence microscopy imaging is rapidly progressing driven by clinical applications and new technologies, respectively.
Susanne K. Golm +2 more
doaj +1 more source
Process Improvement of Adeno-Associated Virus Production
Frontiers in Chemical Engineering, 2022 Adeno-associated viruses (AAVs) have been well characterized and used to deliver therapeutic genes for diseases treatment in clinics and basic research. This study used the triple transient transfection of AAV-DJ/8 as a model expression system to develop
Jia-Shiung Guan +7 more
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
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
Adeno-Associated Virus Vectors and Hematology [PDF]
Blood, 1999 Gene therapists have a special fondness for hematology. Initially, this was due to the many inherited diseases that could potentially be cured by ex vivo genetic modification of hematopoietic stem cells. Once the early murine leukemia virus (MLV) vectors were found to transduce only a small percentage of primate stem cells, the search began for other ...
D W, Russell, M A, Kay
openaire +2 more sources
Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease
Blood Advances, 2017 : Pseudogenes are duplicated genes with mutations rendering them nonfunctional. For single-gene disorders with homologous pseudogenes, the pseudogene might be a target for genetic correction.
Randall K. Merling +14 more
doaj +1 more source
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
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