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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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SRC-3: CoRegen’s revolutionary approach to cancer

Drug Target Review

This interview explores how CoRegen’s innovative techniques are not only transforming cancer treatment but also hold promise for a wide range of medical applications, all while minimising the side effects often associated with traditional therapies. What are the key benefits of your approach?

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Enhancing gene therapy with Circio

Drug Target Review

Secondly, circRNAs can be engineered for more efficient protein expression by carefully selecting and optimising the IRES element, a sequence motif derived from viruses and used to initiate cap-independent translation from circRNA. There are three major reasons for why Circio has selected AATD as its lead gene therapy program.

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Driving efficiency across upstream bioprocess workflow

Drug Target Review

Advancements in genetic and protein engineering through technology innovation, automation, and more recently machine learning, have led to the development of more complex molecules such as bispecific antibodies and antibody drug conjugates. Cell Gene Therapy Insights 2017; 3(2), 131-158, 10.18609/cgti.2017.014. Biotechnol Prog.

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mRNA Cancer Vaccines and Therapies: An Overview

Advarra

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

Vaccine 52
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Codon Digest: CAR-T Therapy for Neuroblastoma

Codon

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. LanzaTech announced a partnership with H&M Move to convert factory carbon emissions into fabrics using engineered microbes. Support for $5 per month. Subscribe to learn more.

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Women in Stem with Dr Beate Mueller-Tiemann

Drug Target Review

A pivotal moment for me was when I realised that if I understood disease mechanisms on a molecular level, I could be part of an industry that helps develop medicines/therapies for those who need them most. Beate is an elected member of the Board of the Dechema, German Society for Chemical Engineering and Biotechnology.