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Researchers devise new way to target and correct disease-related proteins

Broad Institute

Also featured are the FKBP12 binding motif (light blue triangle), the DNA barcode (red double helix), and the combinatorial library element (red hexagon). Related groups Xavier lab Over the past two decades, large genetic studies have linked tens of thousands of DNA variants to thousands of human traits and diseases.

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Scientists create next generation of tools in battle against brain disease

Broad Institute

The team has successfully created over 1000 of them, and each consist of: a harmless adeno-associated virus (or AAV) that acts like a shuttle capable of transporting specially designed DNA into the cell; a segment of DNA (an enhancer) that acts like an activation switch to mark or trigger a change in how the cell functions.

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Scientists develop technology that brings new precision to genome editing

Broad Institute

CRISPR-Cas9, often likened to “molecular scissors,” allows scientists to cut DNA at targeted sites to snip, repair, or replace genes. But despite its power, Cas9 poses a critical safety risk: the active enzyme can linger in cells and cause unintended DNA breaks—so-called off-target effects—which may trigger harmful mutations in healthy genes.

DNA
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New study aims to uncover the genetics of anorexia and other eating disorders

Broad Institute

Researchers at the Broad Institute Ben Neale and Mark Daly are co-leading the study, which allows people across the US to sign up to have their DNA analyzed as part of this effort. The team then sequences the DNA using an approach called the Blended Genome Exome , an efficient and cost-effective alternative to whole-genome sequencing.

DNA
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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

“Unlike gene editing, which makes irreversible DNA changes, engineered tRNA allows for controlled, reversible interventions,” Michelle highlights. “A A single tRNA therapeutic could treat multiple diseases caused by the same mutation.”

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Why More Data Hasn’t Improved Clinical Trial Enrollment

H1 Blog

This paradox, highlighted in a recent Life Sciences DNA podcast featuring Ariel Katz, co-founder and CEO of H1, in conversation with Dr. Amar Drawid, an industry veteran and AI expert, merits deeper exploration for clinical operations professionals seeking to optimize site selection and accelerate patient recruitment. .

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A Visual Guide to Genome Editors

Codon

Certain CRISPR components can add short DNA sequences from the genomes of defeated viruses into the bacterium’s own genome, creating a type of protective “memory.” If the same virus invades the cell a second time, the gRNA’s spacer sequence will bind to the matching viral DNA sequence, then be cut by the Cas protein.

DNA