genetic disease

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Building Bridges for Translational Research - A Special Podcast Series

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Doctors Treat a Rare Genetic Condition Before Patient Is Born

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Scans of Sundry Variant Types Uncover Autism-Linked Genes

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Deficient RNA Editing Implicated in Inflammatory Disease 

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Q&A: Whole Genomes of 150,000 Britons Reveal Novel Genetic Variants

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Physician and Geneticist Leon Rosenberg Dies at 89

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First Person Dosed in Novel Gene Editing Clinical Trial

 Learn how genetics research identifies new targets for complex disease therapies.

Revealing Gene Functions in Health and Disease

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Genetic Mutations Can Be Benign or Cancerous—a New Method to Differentiate Between Them Could Lead to Better Treatments

Optimize quality control in gene therapy viral vector production

Empty or Full: Separating Therapeutically Viable Viral Vectors

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CRISPR-Based Treatment Successfully Lowers Toxic Protein Levels

PATH TO TREATMENT: Marley, pictured here with her father, was diagnosed with the rare genetic condition Bachmann-Bupp syndrome a few years ago.

Doctors Treat Girl’s Genetic Disorder with Repurposed Drug

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Kirsty Wright is searching for evidence in a missing persons investigation. She is wearing a blue shirt and glasses. Behind her is dense vegetation and police procedural tape.

How a Forensic Biologist Exposed a DNA Lab Scandal That Shook Australia

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Universe 25 Experiment

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One Gene Influences 75 Percent of Alzheimer’s Disease Cases

A picture of Rohita Roy, a postdoc at Stanford University.

Postdoc Portrait: Rohita Roy

Multimedia

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From Data to Discovery: Omics in Therapeutic Innovation

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Exploring Organoids for Disease Modeling Research

February 2026

A Stubborn Gene, a Failed Experiment, and a New Path

When experiments refuse to cooperate, you try again and again. For Rafael Najmanovich, the setbacks ultimately pushed him in a new direction.

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Human-Relevant In Vitro Models Enable Predictive Drug Discovery

Advancing Drug Discovery with Complex Human In Vitro Models

Stemcell Technologies
Redefining Immunology Through Advanced Technologies

Redefining Immunology Through Advanced Technologies

Ensuring Regulatory Compliance in AAV Manufacturing with Analytical Ultracentrifugation

Ensuring Regulatory Compliance in AAV Manufacturing with Analytical Ultracentrifugation

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Maximizing Cancer Research Model Systems

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