Tailored nucleases for primary hyperoxaluria

Simon Ng — Thu, 04 Nov 2021 18:04:40 +0000

Devyn Smith, PhD, Arbor Biotechnologies, Cambridge, MA, discusses utilizing tailored nuclease-based CRISPR therapies to treat primary hyperoxaluria, a genetic condition that leads to the accumulation of oxalate in the liver and subsequent end-stage renal disease in children. Current treatments have a mixed efficacy and new genetic therapies aim to treat primary hyperoxaluria by multiplex gene knockout, where the nuclease targets two known protein targets. This interview took place at Meeting on the Mesa 2021.

A novel lentiviral therapeutic approach to primary hyperoxaluria type 1

admin_vjr_user — Fri, 14 May 2021 14:11:24 +0000

Jose-Carlos Segovia, PhD, CIEMAT, CIBERER & IIS- FJD, Madrid, Spain, discusses the limitations associated with the use of adeno-associated virus vectors when targeting primary hyperoxaluria type 1 (PH1), a rare genetic disorder of the liver caused by mutations in the AGXT gene. Given the high integrating capacity of lentiviral vectors, they are being investigated in a preclinical mouse model as a novel therapeutic approach for PH1. Preliminary studies have demonstrated that treatment with hepatocyte-specific lentiviral gene therapy resulted in high levels of gene correction, although further work is required to improve transduction efficiency and enhance therapeutic benefit. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

Primary Hyperoxaluria – VJRegenMed

Tailored nucleases for primary hyperoxaluria

A novel lentiviral therapeutic approach to primary hyperoxaluria type 1