An Universal Gene Therapy for Diamond-Blackfan Anemia Is Poised for Clinical Trials

Treatment designed to work across different genetic mutations At a glance: Efforts to develop a gene therapy for Diamond-Blackfan anemia (DBA) — a rare, life-threatening disorder in which bone marrow cannot make mature, functioning red blood cells — have been hampered by the fact that at least 30 different genetic mutations can cause the disorder.

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Early-stage trial finds stem cell therapy for retinitis pigmentosa is safe

A team of UC Davis Health researchers has shown that CD34+ stem cells can be safely administered into retinitis pigmentosa (RP) patients’ eyes and may offer therapeutic benefits. The phase 1 trial also confirmed that these specialized cells can be readily isolated from the patient’s own bone marrow. The study was published in the American Academy of Ophthalmology journal,

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Novel CAR T cell therapy obe-cel demonstrates high response rates in adult patients with advanced B-cell ALL

Trial results supported recent FDA approval for patients with relapsed/refractory disease Patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (ALL) who were treated with the novel anti-CD19 chimeric antigen receptor (CAR) T cell therapy, obecabtagene autoleucel (obe-cel), experienced high response rates and most did not need a subsequent stem cell transplant (SCT), according to results from the

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New drug provides first boost to asthma attack treatment in 50 years

A team of international researchers has found a drug injected under the skin effectively treats asthma and chronic obstructive pulmonary disease and reduces the need for further treatment. Dr Sanjay Ramakrishnan, from UWA’s Medical School and the Institute for Respiratory Health, was lead author of the paper published in The Lancet Respiratory Medicine. “Asthma and chronic

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Scripps Research scientists identify mutation that could facilitate H5N1 “bird flu” virus infection and potential transmission in humans

New findings underscore the importance of ongoing surveillance for H5N1 mutations that pose risks to public health. vian influenza viruses typically require several mutations to adapt and spread among humans, but what happens when just one change can increase the risk of becoming a pandemic virus? A recent study led by scientists at Scripps Research

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COVID-19 Linked to Long-Term Risk for Autoimmune, Autoinflammatory Disease

Long-term monitoring and management of patients is crucial after COVID-19, considering demographic factors, disease severity, and vaccination status, to mitigate these risks. In a population-based study published in JAMA Dermatology, researchers from the Republic of Korea investigated whether having a history of coronavirus disease 2019 (COVID-19) increased the long-term risk of autoimmune and autoinflammatory connective tissue disorders.

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Space: A new frontier for exploring stem cell therapy

Stem cells grown in microgravity aboard the International Space Station (ISS) have unique qualities that could one day help accelerate new biotherapies and heal complex disease, two Mayo Clinic researchers say.  The research analysis by Fay Abdul Ghani and Abba Zubair, M.D., Ph.D., published in NPJ Microgravity, finds microgravity can strengthen the regenerative potential of cells. Dr.

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Toward better surgical outcomes in patients undergoing knee replacement surgery

Evidence from a questionnaire administered to postoperative patients could settle an ongoing debate regarding best practices in total knee arthroplasty. When performing knee replacement surgery, also called total knee arthroplasty, doctors traditionally try to align the hip, knee, and ankle in a straight line, forming a neutral alignment, rather than replicating the patient’s original alignment.

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