Category REGENERATIVE MEDICINE

New gene therapy treatment resulted in sustained, clinically relevant benefits in children with Metachromatic Leucodystrophy: long term results published.

An investigational gene therapy showed benefit in pediatric patients with early-onset metachromatic leukodystrophy (MLD), a small study showed. Treatment with atidarsagene autotemcel (arsa-cel) preserved motor development and cognitive function in MLD patients enrolled in a prospective phase I/II trial or treated through expanded access programs, reported Alessandro Aiuti, MD, of the San Raffaele Telethon Institute for Gene

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Immunomodulatory effects of mesenchymal stem cells in peripheral nerve injury: a review

Peripheral nerve injury (PNI) refers to varying degrees of trauma to peripheral nerve stems or branches. PNIaccounts for 1.5–4.0% of global trauma cases annually and is one of the most challenging health issues at present. Nerve regeneration is a complicated cellular process involving infammation, neurotrophic factors, neurotransmitters, adhesion, the formation of axons and growth cones,

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Epilepsy Research Boosts Case for New Gene Therapy

Research from the School of Medicine suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition. The gene therapy, developed by Stoke Therapeutics, is now in clinical trials. Because most Dravet syndrome cases are caused by a mutation in the SCN1A gene, resulting in

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Engineered Mesenchymal Stem Cells as a Biotherapy Platform for Targeted Photodynamic Immunotherapy of Breast Cancer

Interleukin-12 (IL12) is a pleiotropic cytokine with promising prospects for cancer immunotherapy. Though IL12 gene-based therapy could overcome the fatal hurdle of severe systemic toxicity, targeted delivery and tumor-located expression of IL12 gene remain the challenging issues yet to be solved. Photo-immunotherapy emerging as a novel and precise therapeutic strategy, which elaborately combines immune-activating agents

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CAR-T-cell therapy has the potential to replace chemoimmunotherapy for second-line treatment of patients with refractory or relapsed large B-cell lymphoma (LBCL)

In the ZUMA-7 trial, at a median follow-up of 24.9 months, patients randomly assigned to receive CAR T-cell therapy with axicabtagene ciloleucel, or axi-cell (Yescarta) had a median event-free survival (EFS) of 8.3 months, compared with 2 months for patients randomly assigned to standard-of-care chemoimmunotherapy, reported Frederick L. Locke, MD, from the Moffitt Cancer Center

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Single injection of messenger RNA Can Be Used to Make CAR T cells in vivo and to attack fibrosis in heart failure .

Penn study reveals an easier, more scalable way to make a powerful immunotherapy An experimental immunotherapy can temporarily reprogram patients’ immune cells to attack a specific target via only a single injection of messenger RNA (mRNA), similar to the mRNA-based COVID-19 vaccines, according to a new study from researchers in the Perelman School of Medicine

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Sloan Kettering Institute Scientists Retool CAR T Cells to Serve as ‘Micropharmacies’ for Cancer Drugs

Immunotherapies called chimeric antigen receptor (CAR) T cells use genetically engineered versions of a patient’s own immune cells to fight cancer. These treatments have energized cancer care, especially for people with certain types of blood cancers. Now, scientists at Memorial Sloan Kettering Cancer Center’s Sloan Kettering Institute (SKI) have developed new CAR T cells that can do something their

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Targeting the fibrin-neutrophil interaction prevented bone loss from periodontal disease

Human and animal study offers insight into treating periodontal disease and other inflammatory disorders. Blocking function of a blood-clotting protein prevented bone loss from periodontal (gum) disease in mice, according to research led by scientists at the National Institute of Dental and Craniofacial Research (NIDCR), part of the National Institutes of Health. Drawing on animal

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CAR-HEMATOTOX score: a model for CAR T-cell–related hematologic toxicity in relapsed/refractory large B-cell lymphoma

Immunotherapy with so-called CAR-T cells has established itself for the treatment of various leukemias (blood cancer) and lymphomas (lymph gland cancer). A team led by Dr. Kai Rejeski and Prof. Marion Subklewe from the Medical Clinic III of the LMU Klinikum found that many patients suffer from a side effect that initially fell under the radar: a

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