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Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Broad Institute

By Broad Communications June 24, 2025 Credit: Casey Atkins David Liu is a core member at the Broad Institute where he is the Richard Merkin Professor and director of the Merkin Institute for Transformative Technologies in Healthcare. Some of these treatments, like K.J.’s, He needed a personalized, one-of-a-kind therapy.

Treatment 133
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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Online January 14, 2025. But the researchers needed to deliver the base editors to the brain. Nature Medicine.

Disease 144
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Gene editing disrupts Huntington’s mutation in mice

Broad Institute

Gene editing disrupts Huntingtons mutation in mice By Allessandra DiCorato May 27, 2025 Breadcrumb Home Gene editing disrupts Huntingtons mutation in mice Making single-letter edits in stretches of repeated DNA stopped or reversed the genetic change that causes Huntingtons disease and Friedreichs ataxia.

DNA 133
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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.

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Cas9 ancestor engineered into a compact genome editing tool

Broad Institute

Cas9 ancestor engineered into a compact genome editing tool By Tom Ulrich May 14, 2025 Breadcrumb Home Cas9 ancestor engineered into a compact genome editing tool Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells. Ten were capable of editing DNA in human cells.

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Analysis Life Sciences Thank You FDA lays out new assessment framework for certain nitrosamines

Agency IQ

EMA’s investigation ended up revealing even more causes of nitrosamine impurities in drugs, discovering, for example , that formation happened during the heat-sealing process of in certain products using blister-packaging. Moving forward, confirmatory testing for approved products should be completed by August 1, 2025.

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Analysis Chemical Thank You What we expect the EPA to do in April 2024

Agency IQ

Important comment periods closing in April Title Type Comments Close Proposed Consent Decree, Clean Air Act Citizen Suit Notice 4/4/2024 Listing of Specific PFAS as Hazardous Constituents Proposed Rule 4/8/2024 Agency Information Collection Activities; Proposed Information Collection Request; Comment Request; Greenhouse Gas Reduction Fund Accomplishment (..)