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A Visual Guide to Gene Delivery

Codon

A gene is the essential hereditary unit that passes traits from parents to offspring; a segment of DNA containing instructions for making a specific protein or molecule that performs a particular function in your body. As methods for resolving errors in DNA continue to advance, it has never been easier to fix a broken gene.

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Prime editing treats childhood brain disease in mice

Broad Institute

Prime editing was developed in 2019 by the lab of David Liu , a Broad core institute member and co-senior author of the new paper. They demonstrated that they could correctly repair AHC mutations in up to 90 percent of treated cells, with minimal changes to other stretches of DNA.&

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FDA Eliminates Animal Testing: Impact on Biotechs

The Premier Consulting Blog

In 2019, we published a detailed peer-review article outlining this process based on our long history of leveraging existing animal and human data to streamline drug development programs and reduce or eliminate animal testing under the 505(b)(2) pathway.[ 3] Salminen et al. link] [7] H.R.2565

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Prime Editing: Adding Precision and Flexibility to CRISPR Editing

addgene Blog

But prime editing, a CRISPR technique developed in David Lius lab in 2019, has added considerably more precision and flexibility to the CRISPR editing world. All of this is possible without double-strand breaks (DSBs) or donor DNA templates. Plus, it can combine different types of edits with one another.

DNA
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Tofersen

New Drug Approvals

Liu A (1 May 2019). Jump up to: a b New Drug Therapy Approvals 2023 (PDF). Food and Drug Administration (FDA) (Report). January 2024. Archived from the original on 10 January 2024. Retrieved 9 January 2024. ^ “Biogen’s antisense ALS drug shows promise in early clinical trial” FierceBiotech. Retrieved 25 April 2023.

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Altasciences at ASGCT 2025: An Open Forum for All Things Gene and Cell Therapy | By Kaylyn Koenig

Alta Sciences

I also walked attendees through some of our DNA and RNA isolation techniques and our molecular biology laboratory’s experience in collecting samples and isolating genetic material to assess germline integration after preclinical studies.

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A Visual Guide to Genome Editors

Codon

” Four years earlier, in 2019, Gray had become the first patient with sickle cell anemia — a genetic disorder that causes red blood cells to become sticky and rigid — to receive an experimental treatment using CRISPR genome editing. While crossing Trafalgar Square, Gray paused briefly to reflect on her experience.

DNA