Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues. Recombinan
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to the growt...
Affinia Therapeutics is expanding what's possible in adeno-associated virus (AAV) gene therapy today and bringing transformative medicines for people.
Adeno-associated virus(AAV) is a nonenveloped,ssDNAvirus in theparvovirusfamily, which has become one of the leading candidate vectors for human gene therapy.AAVhas been studied extensively to identify host cellular factors involved in infection, as well as to identify capsid variants that confer ...
Severaltrialswith AAV are on-going or in preparation, mainly trying to treat muscle and eye diseases; the two tissues where the virus seems particularly useful. However, clinical trials have also been initiated where AAVvectorsare used to deliver genes to the brain. This is possible because AAV...
Adeno-associated virus (AAV) is one of the most actively investigated gene therapy vehicles. It was initially discovered as a contaminant of adenovirus preparations [4,5], hence its name. Simply put, AAV is a protein shell surrounding and protecting a small, single-stranded DNA genome of appro...
(cell therapy), Adeno-Associated Viruses (AAV) have strengthened their position as a leading platform for gene delivery especially after recent landmark approvals: Luxturna (voretigene neparvovec), an AAV-based therapy that delivers a functional copy of the RPE65 gene to pat...
Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues. Recombinant AAV (rAAV) has been engineered for enhanced specificity and developed as a tool for...
Consequently, Qiao et al20 conducted an adeno-associated virus (AAV)-mediated gene therapy approach to assess the therapeutic potential of mouse mAgrin in the treatment of mice with LAMA2 deficiencies. In their study, they demonstrated that overexpression of the mAgrin ameliorated muscle structural ...
(genus Dependovirus of the family Parvoviridae) that enter the nucleus of a host cell but are dependent on coinfection with an adenovirus or herpesvirus for their replication, that infect a wide range of hosts but do not appear to cause disease, and that are used in gene therapy as ...