Human Immunodeficiency Virus – VJRegenMed

HSC gene editing: current approaches and remaining challenges

admin_vjr_user — Thu, 19 Aug 2021 11:17:13 +0000

Valentina Vavassori, PhD, IRCCS San Raffaele Scientific Institute & Vita-Salute San Raffaele University, Milan, Italy, provides an overview of the current landscape for hematopoietic stem cell (HSC) gene editing and describes key gene editing approaches used including the non-homologous end joining (NHEJ) and homology-directed repair (HDR) pathways. Key lines of ongoing research to advance HSC gene editing approaches include the improvement of HDR efficiency in HSCs, as well as the development of new tools, such as CRISPR-associated transposases, to circumvent toxicity associated with double-strand breaks. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

Developing CAR T-cell therapy for HIV

admin_vjr_user — Fri, 13 Aug 2021 17:10:39 +0000

Marco Alessandrini, PhD, Antion Biosciences SA, Geneva, Switzerland, discusses the development of an allogeneic chimeric antigen receptor (CAR) T-cell therapy for HIV. Antion’s novel miCAR platform was used to generate a bimodal gene construct that expressed both an HIV-specific CAR with a truncated CD4 receptor and a microRNA-based gene silencing cassette to silence CCR5, the T-cell receptor and inhibitory receptors, in order to enhance CAR T-cell persistence. This work highlights the potential to develop a scalable, cost-effective platform for creating off-the-shelf, universally accessible CAR-T therapy for HIV. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.