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A team of researchers at Children's Hospital of Philadelphia (CHOP), in collaboration with Beam Therapeutics, tested and developed an "off-the-shelf" chimeric antigen receptor T-cell (CART) using base editing technolo.
CAR-T cell therapy has revolutionized the treatment of relapsed or refractory blood cancers, with several products authorized by the Food and Drug Administration (FD.
To address these obstacles, researchers have used genome editing techniques to modify allogeneic CART products, but many of these tools -- such as CRISPR-Cas9 -- rely on DNA double-strand breaks (DSBs), which can lead to unexpected results and potential unforeseen consequenc.
Base editing is an emerging technology that enables high-precision and high-efficiency changes to individual base pairs at specific poin.
To determine whether gene editing to generate a CART is clinically feasible, CHOP researchers, led by .
In multiple in vivo and in vitro preclinical studies, researchers observed that 7CAR8 did not affect T-cell proliferation or cause genomic rearrangement produc.
"Laboratory studies have shown that 7CAR8 is highly active in preclinical models and, if approved, could potentially be a treatment for children and adults with relapsed or refractory T-A.
