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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. Credit: Neumann EN, Bertozzi TM, et al.

Disease 142
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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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Bayer and Broad Institute extend cancer therapy research collaboration

Broad Institute

Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.

Therapies 105
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Scaling Phage Therapy

Codon

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Goodsell, RCSB Protein Data Bank and Scripps Research. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Always free.

Therapies 117
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New gene therapy for muscular dystrophy offers hope

Science Daily: Pharmacology News

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

Therapies 124
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Immuno-oncology innovations redefining cancer treatment in 2024

Drug Target Review

What innovative approaches and technologies are anticipated to reshape immuno-oncology treatments in 2024? Immuno-oncology, notably through checkpoint inhibitors, has significantly reshaped cancer treatment over the past decade.

Treatment 122
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Unlocking the power of stem cell therapy

Drug Target Review

What key findings about stem cell behaviour, differentiation and integration within host tissues impact the development of stem cell therapies? Despite their potential, ADSC therapy faces several challenges in preclinical studies. Is there a certain disease or condition that you believe stem cell therapy holds the most promise for?

Therapies 105