Results 21 to 30 of about 6,309 (150)

Molecular mechanisms of amyotrophic lateral sclerosis as broad therapeutic targets for gene therapy applications utilizing adeno‐associated viral vectors

open access: yesMedicinal Research Reviews, Volume 43, Issue 4, Page 829-854, July 2023., 2023
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

open access: yesTransboundary and Emerging Diseases, Volume 2023, Issue 1, 2023., 2023
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

open access: yesArquivos de Neuro-Psiquiatria, 2022
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

open access: yesResearch and Practice in Thrombosis and Haemostasis, Volume 6, Issue 4, May 2022., 2022
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

Advancements in molecular design and bioprocessing of recombinant adeno‐associated virus gene delivery vectors using the insect‐cell baculovirus expression platform

open access: yesBiotechnology Journal, Volume 16, Issue 4, April 2021., 2021
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

Construcción de un vector de expresión derivado de virus adenoasociados para corregir in vitro el defecto genético de la enfermedad de Morquio A

open access: yesBiomédica: revista del Instituto Nacional de Salud, 2008
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]

open access: yes, 2015
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

A sensitive and reproducible cell-based assay via secNanoLuc to detect neutralizing antibody against adeno-associated virus vector capsid

open access: yesMolecular Therapy: Methods & Clinical Development, 2021
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  

The seroprevalence of neutralizing antibodies against the adeno-associated virus capsids in Japanese hemophiliacs

open access: yesMolecular Therapy: Methods & Clinical Development, 2022
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]

open access: yes, 2008
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

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