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New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If

Therapies 137
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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

Disease 142
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Scaling Phage Therapy

Codon

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

Therapies 120
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OBN Invites Life Science Companies, Industry Leaders and Investors to BioTrinity 2022

Drug Discovery Today

• Face-to-face event, taking place in London, UK, 26-27th April, will feature two dedicated tracks: ‘Scientific’, focused on seven core therapy areas and ‘Life Sciences Business’ Programme features keynote from George Freeman MP, with an update on the UK Government Life Sciences VisionRegistration for BioTrinity 2022 now available online

Science 113
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Macrophage cell therapy: a new hope for chronic liver disease patients

Drug Target Review

Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?

Therapies 116
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100x improvement in sight seen after gene therapy trial

Science Daily: Pharmacology News

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

Therapies 126
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CAR-NK cells: promising for cancer therapy

Drug Target Review

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

Therapies 118