Allogeneic hematopoietic stem-cell transplantation for X-linked severe combined immunodeficiency (SCID-X1) often fails to reconstitute immunity associated with T cells, B cells, and natural killer (NK ...
SAN DIEGO -- Results from a small single-center study showed that treatment with a gene therapy involving the use of lentiviral vector-transduced autologous hematopoietic stem cells (HSCs) increased ...
The DNA-repair enzyme Artemis is essential for rearrangement of T- and B-cell receptors. Mutations in DCLRE1C, which encodes Artemis, cause Artemis-deficient severe combined immunodeficiency (ART-SCID ...
As more cell, gene and immunotherapy applications emerge for lentiviral vectors, demand for their production continues to rise. With its move last week into a larger manufacturing facility, CDMO ...
Seventeen patients with R/R MCL received a single dose of LV20.19 CAR T cells at 2.5 × 10 6 cells/kg (phase I = three patients; phase II = 14 patients). The best overall response rate (ORR) was 100% ...
After an investigation in an adverse event, Bluebird Bio announced that it is “very unlikely” that the BB305 lentiviral vector (LVV) caused the case of acute myeloid leukemia (AML) which was reported ...
According to the Pharmaceutical Research and Manufacturers of America (PhRMA), some 362 cell and gene therapies are now in clinical development in the United States, up from 289 in 2018. 1 Although ...
RNA interference is a biological process that has evolved with the evolution of mammals and plays an important role in transient and long-term blocking of protein expression. Short Hairpin RNA (shRNA) ...
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