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Genespire and SR-TIGET Show Durable Preclinical Efficacy of Liver-Directed Gene Therapy for Methylmalonic Acidemia
Milan, Italy, July 08 — Genespire, in collaboration with researchers at the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), has today announced the publication of preclinical data supporting the potential of its liver-directed immune-shielded lentiviral gene therapy approach to treat methylmalonic acidemia (MMA), a severe inherited metabolic disorder. The findings, published in the Journal of Hepatology, show that a single systemic administration of a lentiviral vector encoding the MMUT gene led to sustained improvements in disease features in a validated mouse model of MMA, with effects lasting for the average lifespan of laboratory mice. Since the mice were treated when young, the study supports the durability of the gene therapy through postnatal growth and maturation of the liver. MMA is caused by a deficiency of methylmalonyl-CoA mutase, an enzyme that plays a critical role in the body’s metabolism of food. Its absence results in the accumulation of toxic metabolites that lead to recurrent metabolic crises, growth failure, neurological impairment, and multi-organ damage. In the study, researchers also treated mice with a dose containing an optimized MMUT transgene, thereby improving therapeutic efficacy. In the same mouse model of MMA, this…
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MP Expands Digital Healthcare Reach Under Ayushman Bharat Mission
Bhopal, July 8: The Madhya Pradesh Government is working to expand digital healthcare services to the remotest parts of the…
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IIM Lucknow Study Identifies Key Factors for Strengthening India’s Medical Tourism Appeal
Lucknow, July 8: Researchers from Indian Institute of Management Lucknow and Fortis Hospital, Delhi, have conducted a study examining how cultural sensitivity, communication, and expectation management…
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Cashless Healthcare Scheme Brings New Medical Benefits for UP Teachers and Education Staff
Lucknow, July 8: The Uttar Pradesh government has launched a new cashless healthcare scheme aimed at providing easier and more…
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Navi Mumbai Airport Approved for Import of Medicines
New Delhi, July 8: The Government has approved Navi Mumbai International Airport as an authorised point for importing medicines into…
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Gujarat Health Centres Clear 5 Lakh Unused Items in Cleanliness Drive
Gandhinagar, July 7: Health facilities across Gujarat have removed more than five lakh unused and obsolete items as part of…
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ABDM Crosses 104 Crore Linked Health Records Milestone
July 6: The Ayushman Bharat Digital Mission (ABDM) has achieved a major milestone by linking over 104 crore health records across…
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Dr. Smiti Jassar Klaire Assumes Advisory Board Chair to Advance Founder’s Vision
Modinagar, July 6 : Nearly three decades ago, Late Shri Ajit Singh Jassar established the Jassar Dental Medical Education Health Foundation with…
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Himalaya Wellness Reimagines Hydration Skincare with the Relaunch of Its Aloe Range Through a Youthful TVC
~The new “Say Aloe to Hydrated, Bouncy, Dewy Cheeks” campaign spotlights hydration through nature enriched with science. Hyderabad, July 4:Himalaya…
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NITI Aayog Plans Global Licensing Push for Ayurveda
July 3: NITI Aayog has proposed a global licensing framework to help standardise Ayurveda practices and products and expand their…