ASGCT 2021: Time to celebrate and expand.

Mol Ther

Executive Editor of the Molecular Therapy family of journals, Molecular Therapy, Seattle, WA, USA.

Published: July 2021

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8261252PMC
http://dx.doi.org/10.1016/j.ymthe.2021.06.011DOI Listing

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On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in preclinical and clinical AAV gene therapy, to further contextualize and understand this phenomenon. Recombinant AAV (rAAV) vectors are used to develop therapies for many conditions given their ability to transduce multiple cell types, resulting in long-term expression of transgenes. Although most rAAV DNA typically remains episomal, some rAAV DNA becomes integrated into genomic DNA at a low frequency, and rAAV insertional mutagenesis has been shown to lead to tumorigenesis in neonatal mice.

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ASGCT 2021: Time to celebrate and expand.

Mol Ther

July 2021

Executive Editor of the Molecular Therapy family of journals, Molecular Therapy, Seattle, WA, USA.

View Article and Find Full Text PDF
Article Synopsis
  • - Gene therapy is a promising option for treating genetic diseases, particularly monogenic metabolic disorders, where liver transplantation is usually the only solution.
  • - The liver's significant blood volume capacity makes it an ideal site for protein secretion, and advancements in gene delivery methods have emerged, especially targeting liver cells (hepatocytes) through both viral and increasingly popular non-viral vectors.
  • - The review discusses various strategies involving gene addition and editing, highlights the use of RNA as a therapeutic tool, and examines the limitations of current liver-targeted gene therapies along with potential solutions.
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