Results 21 to 30 of about 6,309 (150)
Abstract Despite the devastating clinical outcome of the neurodegenerative disease, amyotrophic lateral sclerosis (ALS), its etiology remains mysterious. Approximately 90% of ALS is characterized as sporadic, signifying that the patient has no family history of the disease.
Jessica Merjane+3 more
wiley +1 more source
The Analysis of Oral and Fecal Virome Detects Multiple Novel Emerging Viruses in Snakes
Wild animals are considered reservoirs for emerging and reemerging viruses, such as the novel severe acute respiratory syndrome coronavirus 2 (SARS‐CoV‐2). Previous studies have reported that bats and ticks harbored variable important pathogenic viruses, some of which could cause potential diseases in humans and livestock, while viruses carried by ...
Aijing Liu+13 more
wiley +1 more source
Gene therapy in neuromuscular disorders
Monogenic neuromuscular disorders are potentially treatable through gene therapy. Using viral vectors, a therapeutic transgene aims to restore normal levels of a protein not produced by the defective gene, or to silence a gene whose expression leads to ...
Rodrigo Holanda Mendonça+1 more
doaj +1 more source
Seroprevalence to adeno‐associated virus type 6 in people with hemophilia B from a UK adult cohort
Abstract Background Gene therapy shows promise as a potential “cure” for hemophilia A and B. Adeno‐associated virus (AAV) vectors are the leading platform to deliver modified genetic code of factor VIII or IX to the liver effecting endogenous production. Patient exposure to wild‐type AAV leads to the formation of neutralizing factors, which can prevent
Sara Boyce+9 more
wiley +1 more source
This review focus on the advancements of the molecular design of baculovirus vectors for high yield production of adeno‐associated virus (AAV) vectors for gene delivery describing the different generation of expression vectors with associated advantages and critical limitations.
Pranav R.H. Joshi+3 more
wiley +1 more source
Introducción. La mucopolisacaridosis IV A (Morquio A) es una enfermedad de depósito lisosómico causada por la deficiencia en la actividad de la enzima N-acetil-galactosamina- 6-sulfato-sulfatasa que produce la acumulación intralisosómica de queratán y ...
Luis Alejandro Barrera+6 more
doaj +1 more source
Novel parvoviruses in reptiles and genome sequence of a lizard parvovirus shed light on Dependoparvovirus genus evolution. [PDF]
Here, we report the detection and partial genome characterization of two novel reptilian parvoviruses derived from a short-tailed pygmy chameleon (Rampholeon brevicaudatus) and a corn snake (Pantherophis guttatus) along with the complete genome analysis ...
Benkő, Mária+3 more
core +3 more sources
Most gene therapy clinical trials that systemically administered adeno-associated virus (AAV) vector enrolled only patients without anti-AAV-neutralizing antibodies.
Nemekhbayar Baatartsogt+6 more
doaj
Adeno-associated virus (AAV) vectors are promising modalities of gene therapy to address unmet medical needs. However, anti-AAV neutralizing antibodies (NAbs) hamper the vector-mediated therapeutic effect.
Yuji Kashiwakura+29 more
doaj
Identification of novel porcine and bovine parvoviruses closely related to human parvovirus 4 [PDF]
Human parvovirus 4 (PARV4), a recently discovered parvovirus found exclusively in human plasma and liver tissue, was considered phylogenetically distinct from other parvoviruses.
Au, WK+18 more
core +1 more source