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FDA Returns Disappointing News for ALS Stem Cell Therapy

PLOS: DNA Science

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). It aims to correct the faulty instructions at the DNA level, straightforward, at least conceptually, because mutations are deletions, of part or all of the gene. That’s not the case for ALS.

Therapies 101
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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

Related groups Liu Group Related news New CRISPR genome editing system offers a wide range of versatility in human cells Researchers extend power of gene editing by developing a new class of DNA base editors In May 2025, researchers announced that K.J. While doctors are cautious about using the word "cure" so early, it is a triumph for K.J.'s

Treatment 133
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Scaling Phage Therapy

Codon

Some algorithms simply look for important similarities between phage and bacterial DNA sequences, a signal of the extensive gene-swapping that goes on when a particular virus and bacteria have shared history. Middle and Right) Once a bacteriophage lands on a cell, it injects its DNA through a tube (purple) into the unwilling host.

Therapies 137
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Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

Lumping gene and cell therapies together isn’t very helpful – the cell-based ones are mostly doctored T cells to treat cancer. The package insert for the drug, Elevidys , warns of adverse effects of acute liver injury and inflammation around the heart and muscles. million DNA bases. appeared first on DNA Science.

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Codon Digest: Seeing Colors After Gene Therapy

Codon

There’s a TV show where a bunch of doctors are walking through a hospital corridor. They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. Transcription factors are proteins that bind to DNA and control gene expression. From McKyton et al.

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Codon Digest: Seeing Colors After Gene Therapy

Codon

There’s a TV show where a bunch of doctors are walking through a hospital corridor. They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. Transcription factors are proteins that bind to DNA and control gene expression. From McKyton et al.

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Which Arthritis Painkiller Works the Fastest?

The Pharma Data

WITHOUT doctors, physical therapists or chiropractors. Our Unique Solution Has Been Featured All Over The Media, From Hallmark, The Doctors, Atlanta Live & Arizona Midday…. I Have A Doctorate In Pharmacy, But Everything I Learned About Pain Relief In Pharmacy School Is Dead Wrong! And then got my Doctor of Pharmacy (Pharm.