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#ScienceSaturday: March 2, 2024

KIF1A

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Studies like SPARK highlight the importance of human data in accelerating patient-facing research. Want to learn more about translational research?

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Condensate biology: advancing drug discovery for complex diseases

Drug Target Review

In recent years, a novel approach known as condensate biology has emerged, revolutionising the way researchers think about drug discovery and development. Trained as a physician scientist, he obtained his MD/PhD from Cornell Rockefeller and Sloan Kettering, specialising in Immunology, Genetics, and Cancer Biology.

Disease 98
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A Defense of Peer Review

Codon

He suggests that peer review doesn’t “do the thing it’s supposed to do” — namely, “catch bad research and prevent it from being published,” so perhaps scientists would be better off uploading PDFs to the internet where review and oversight can happen post-publication.

Science 110
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Optimising CRIPSR gene editing of hard-to-transfect cells

Drug Target Review

Controlling the amount of RNP entering each cell is challenging, leading to uncertainty about the ratio of RNP that reaches any specific cell. Importantly for the researcher, there is no assurance that the RNP, once inside the cells, will reliably enter their nuclei.

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Optimising CRISPR gene editing of hard-to-transfect cells

Drug Target Review

Controlling the amount of RNP entering each cell is challenging, leading to uncertainty about the ratio of RNP that reaches any specific cell. Importantly for the researcher, there is no assurance that the RNP, once inside the cells, will reliably enter their nuclei.

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Building better brain models for Parkinson’s disease and beyond

Drug Target Review

Turning this tide, Professor Jens Christian Schwamborn, a cell biology expert at the University of Luxembourg and co-founder of OrganoTherapeutics , is leading a groundbreaking effort to transform our understanding and treatment of these debilitating diseases. Schwamborns research focuses on creating patient-specific brain organoids.

Disease 52
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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. Over the course of 12 years, the Walter Reed Army Institute of Research (WRAIR) screened more than 250,000 compounds in search of an efficacious treatment.

Drugs 139