Category REGENERATIVE MEDICINE

PIER MARIA FORNASARI
2021-12-16

Blockade or deletion of IFN-gamma reduces macrophage activation without compromising CAR T function in hematologic malignancies

Blood Cancer Discovery, a journal of the American Association for Cancer Research, has published a research article demonstrating a novel approach that may reduce a serious adverse effect associated with chimeric antigen receptor (CAR) T-cell therapy, a form of immunotherapy. This research was presented at the American Society of Hematology Annual Meeting in Atlanta, Georgia

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PIER MARIA FORNASARI
2021-12-16

Dual-vector gene therapy restores cochlear amplification and auditory sensitivity in a mouse model of DFNB16 hearing loss

Scientists at Boston Children’s Hospital have successfully used gene therapy to reverse a form of genetic hearing loss in mice. The team corrected a gene mutation that affects sensory hair cells in the inner ear, and the new jigsaw-like method could help improve gene therapy for other disorders. The target of the new treatment was

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PIER MARIA FORNASARI
2021-12-11

Potentially Serious Side Effect Seen in Patient after CAR-T cell treatment for multiple myeloma

Mount Sinai scientists have become the first to report a potentially serious side effect related to a new form of immunotherapy known as CAR-T cell therapy, which was recently approved for the treatment of multiple myeloma. Their findings were published as a case study in Nature Medicine in December. Multiple myeloma is a complex and incurable type

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PIER MARIA FORNASARI
2021-12-10

New Technology published by Caltech researchers is One Step Closer to Targeted Gene Therapy

Broad and robust transgene expression across brain regions of marmoset is shown after systemic delivery with engineered capsid. Gene therapy is a powerful developing technology that has the potential to address myriad diseases. For example, Huntington’s disease, a neurodegenerative disorder, is caused by a mutation in a single gene, and if researchers could go into

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PIER MARIA FORNASARI
2021-12-10

Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye disease.

Clinical trial planning underway to test the widely used diabetes drug metformin as a preventive treatment for the blinding eye disease Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye disease. Researchers from the National Eye Institute (NEI), part of

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PIER MARIA FORNASARI
2021-12-03

Robotic high-throughput biomanufacturing and functional differentiation of human pluripotent stem cells using the CompacT SelecT (CTST) platform

The CompacT SelecT (CTST) platform is a modular robotic system that integrates a full range of cell culture proceduresunder sterile conditions that mimic the manual cell culture process. These procedures include automated handling of different cell culture vessels, pipetting large and small volumes at adjustable speeds, cell counting, cell viability analysis, cell density assessment, microscopic

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PIER MARIA FORNASARI
2021-12-01

A new CRB Life Sciences Report Highlights rapid advances in the development of cell and gene therapies

Rapid advances in the development of cell and gene therapies over the past several years have heralded “an evolutionary time in medicine.” That’s the opinion of Peter Walters, Director of Advanced Therapies at CRB. That thought reflects data seen in CRB’s annual Horizon Life Sciences report that highlights the rapid expansion of the development of cell and

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PIER MARIA FORNASARI
2021-11-30

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A in 16 out of 18 volunteers

About 20,000 people in the U.S. live with hemophilia A. It’s a rare X-linked genetic disorder that affects predominantly males and causes their blood to clot poorly when healing wounds. For some, routine daily activities can turn into painful medical emergencies to stop internal bleeding, all because of changes in a single gene that disables

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PIER MARIA FORNASARI
2021-11-18

Gene therapy effective in children with severe Hurler’s syndrome: the treatment, developed by SR-Tiget in Milan published in NEJM

The first tests on eight children show favorable preliminary results, with an adjustment of motor and cognitive skills and improvements in some brain and joint parameters Gene therapy takes steps forward in the fight against rare diseases. Eight children with severe Hurler’s syndrome, a disease associated with reduced life expectancy, benefited from this therapy. The

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PIER MARIA FORNASARI
2021-11-17

Incidence and risk factors associated with bleeding and thrombosis following chimeric antigen receptor T-cell therapy

Chimeric antigen receptor T-cell (CAR T) therapy has revolutionized the field of malignant hematology. CD19-targeted CAR T-cell therapy has resulted in effective, often durable, responses for chemotherapy-refractory B-cell lymphoma and B-acute lymphoblastic leukemia (B-ALL) in prospective clinical trials. CAR T cells against B-cell maturation antigen have also shown overall response rates in the range of

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