Valentina Vavassori – 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.

T cell and HSC gene editing for the treatment of Hyper-IgM Type 1

admin_vjr_user — Wed, 18 Aug 2021 16:32:44 +0000

Valentina Vavassori, PhD, IRCCS San Raffaele Scientific Institute & Vita-Salute San Raffaele University, Milan, Italy, describes a gene editing approach under investigation for the treatment of hyper IgM Type 1 (HIGM1), an immunodeficiency disorder caused by mutations in the gene for CD40L, a T-cell surface molecule. The strategy involves the insertion of a 5’-truncated corrective CD40L cDNA into the native gene, which restores gene function and maintains the physiological regulation of the gene. This editing strategy was applied to both autologous T cells and hematopoietic stem cells (HSCs), which were then transplanted into mouse models to compare therapeutic efficacy of the two cell types. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

HSC gene editing highlights from ASGCT 2021

admin_vjr_user — Wed, 18 Aug 2021 16:32:42 +0000

Valentina Vavassori, PhD, IRCCS San Raffaele Scientific Institute & Vita-Salute San Raffaele University, Milan, Italy, discusses her highlights from the American Society for Cell & Gene Therapy (ASGCT) Annual Meeting 2021. Exciting advances in gene editing technology include the development of CRISPR-associated transposase systems and prime editing, which can overcome toxicities associated with double-strand breaks in hematopoietic stem cells. In vivo genome editing is another promising area of research, despite safety and efficacy challenges that still need to be addressed before this technology can be used in the clinic. Dr Vavassori also highlights ongoing research into the potential of adverse events and toxicity caused by genome editing, including chromothripsis and translocations, as gene therapies approach clinical approval. This interview took place during the ASGCT 24th Annual Meeting 2021.