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How to advance AAV-based gene therapies

Drug Discovery World

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

Therapies 163
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A New Type of Bookworm: Bringing Patient Perspectives Into the Classroom?

KIF1A

In this blog post, KIF1A.ORG Chief Science Officer Dr. Dominique Lessard describes a unique cross disciplinary experience with multiple scientific stakeholders in the rare disease space, all focused around our holistic understanding of KAND. How do we move the needle to make patient voices and experiences the center of scientific discovery?

Disease 98
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Who are the AACR Scientific Achievement Award winners?

Drug Discovery World

The American Association for Cancer Research (AACR) will honour the following cancer researchers, physician-scientists, advocates, and journalists during its annual meeting to be held April 14-19 at the Orange County Convention Center in Orlando, Florida. June’s award lecture will be held on Sunday April 16 at 4:30pm ET.

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Women in Stem with Dr Sara Sangha

Drug Target Review

Early in my education, I developed an interest in understanding how chemical entities derived from natural resources could be modified and used to fight specific diseases. This resulted in many research fellowship programs not providing leadership opportunities to women due to a fear that the women would eventually quit.

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The promise of 3A5C7 mAb in the treatment of morphine tolerance

Drug Target Review

Considering the translational potential of this research to clinical applications, what are the key preclinical findings that present the 3A5C7 monoclonal antibody as a promising therapeutic option for managing morphine tolerance in humans?

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A research team searches for every gene that helps tumors evade immunotherapy

Broad Institute

By Allessandra DiCorato October 11, 2023 In 2011, Robert Manguso was working in a cell biology lab when his mother was diagnosed with Merkel cell carcinoma, a rare and aggressive skin cancer. His mother had a presentation of the disease that suggested her immune system was already on the job.

Research 137
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Discovering an Antimalarial Drug in Mao’s China

Codon

Chinese researchers scoured ancient medical texts containing written records of malaria outbreaks and the plant-derived recipes that were used to treat them nearly two thousand years ago. “Cumulatively the deadliest of the human infectious diseases,” malaria has been documented throughout recorded history.

Drugs 141