Category REGENERATIVE MEDICINE

Impact of high-risk cytogenetics on outcomes for children and young adults receiving CD19-directed CAR T-cell therapy

CD19 CAR T-cell therapy is effective at achieving durable remission for relapsed/refractory ALL across cytogenetic risk groups. CD19 CAR T-cell treatment results for patients with high-risk cytogenetics including Ph+, Ph-like, and KMT2A-rearranged ALL are encouraging. Chimeric antigen receptor (CAR) T-cell therapy can induce durable remissions of relapsed/refractory B-acute lymphoblastic leukemia (ALL). However, case reports suggested differential

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A Mount Sinai-led team developed a reproducible and scalable method to advance maturation of human pluripotent stem cell-derived cardiomyocytes (hPSC-CMs)

A study reporting this new protocol was published in the April 7 print edition of the journal Cell Stem Cell. A Mount Sinai-led team has developed a reproducible and scalable method to advance maturation of human pluripotent stem cell-derived cardiomyocytes (hPSC-CMs)—cells that support heart muscle contraction, generated in the lab from human stem cell lines—which researchers

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Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

BACKGROUND Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter. The efficacy and safety of the therapy were previously evaluated in men with severe hemophilia A in a phase 1–2 dose-escalation study. METHODS Was conducted an open-label, single-group, multicenter, phase 3

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CAR T cells expressing a pluripotent pro-inflammatory neutrophil-activating protein (NAP) from Helicobacter pylori to better fight cancer

Immunotherapy is increasingly becoming a successful way to treat cancer. Researchers at Uppsala University have now developed armed CAR-T cells that reinforce the immune defense against cancer and that could increase the possibilities to successfully treat solid tumors. The study has been published in the journal Nature Biomedical Engineering. The use of immunotherapy to treat cancer

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Specialized Liver Blood Vessel Identity Factor Required for Regeneration

Researchers at Weill Cornell Medicine have identified a key protein that induces the program to build specialized liver blood vessels. The discovery could lead to engineered replacement hepatic tissue to treat common liver diseases. There are many types of blood vessels in the human body that are functionally different from each other. In the liver,

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In animal study, implant churns out CAR-T cells to combat cancer

Researchers from North Carolina State University and the University of North Carolina at Chapel Hill have developed an implantable biotechnology that produces and releases CAR-T cells for attacking cancerous tumors. In a proof-of-concept study involving lymphoma in mice, the researchers found that treatment with the implants was faster and more effective than conventional CAR-T cell

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