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Prime editing efficiently corrects cystic fibrosis mutation in human lung cells

Broad Institute

Next, researchers will need to develop ways to package and deliver the prime editing machinery to the airways in mice and ultimately humans. Prime editing, a more flexible and controlled kind of gene editing that does not require double-stranded breaks, could help address this limitation. The new approach also generated 3.5

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Analytical Testing Considerations for Gene Therapy Products

The Premier Consulting Blog

The analytical package, consisting of release, stability, and characterization tests, includes data generated throughout the product development and manufacturing process. A more rigorous identity test may sometimes be required, such as DNA sequencing of the entire AAV genome.

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DNA Analysis Finds New Target for Diabetes Drugs

NIH Director's Blog: Drug Development

Credit: Jane Ades, National Human Genome Research Institute, NIH Type 2 diabetes (T2D) tends to run in families, and over the last five years the application of genomic technologies has led to discovery of more than 60 specific DNA variants that contribute to risk.

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Unveiling neoantigen-directed cancer treatment

Drug Target Review

As part of the TCR discovery process and preclinical data package, Adaptive performs rigorous functional characterisation and safety assessments of potent, therapeutic grade antigen-specific TCRs. While tumour cells share a majority of their DNA with healthy cells, they also carry numerous unique mutations.

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

PLOS: DNA Science

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. The DMD gene therapy delivers a shortened version of the dystrophin gene, just 4,558 DNA bases. appeared first on DNA Science. Muscles stop working.

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Enhancing gene therapy with Circio

Drug Target Review

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? DNA vectors in mouse models? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context.

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AI-designed protein awakens silenced genes, one by one

The Pharma Data

The new technique controls gene activity without altering the DNA sequence of the genome by targeting chemical modifications that help package genes in our chromosomes and regulate their activity. In this experiment, however, the cutting function of the Cas9 protein is disabled so the genomic DNA sequence is unaltered.

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