Remove 2022 Remove DNA Remove Immune Response Remove Packaging
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Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

Can they deliver healing genes without triggering an overactive immune response? The details are disturbingly reminiscent of the famous case of Jesse Gelsinger , who died from a ferocious immune response to experimental gene therapy in September 1999. million DNA bases. Muscles stop working.

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Codon Digest: Seeing Colors After Gene Therapy

Codon

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. billion units of DNA code are transcribed into more than a hundred volumes, each a thousand pages long, in type so small as to be barely legible.” a lower risk of immune responses.

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Codon Digest: Seeing Colors After Gene Therapy

Codon

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. billion units of DNA code are transcribed into more than a hundred volumes, each a thousand pages long, in type so small as to be barely legible.” a lower risk of immune responses.

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Optimising AAV capsid purification through improved analytics

Drug Discovery World

This helps ensure a high concentration of loaded capsids and adequate removal of impurities, such as host cell proteins and DNA. In AAV therapeutics, empty capsids are those that are not packaged with the therapeutic DNA. DDW Volume 24 – Issue 1, Winter 2022/2023 References: Wright JF. 2008;15(11):840-848. doi:10.1038/GT.2008.65

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Article FDA Thank You Draft guidance on potency assays for CGT products garners extensive stakeholder input

Agency IQ

For therapeutic protein products , a critical use of assays is the detection and assessment of potential anti-drug antibody (ADA) formation, signaling a product’s potential to induce an unwanted immune response. Additionally, six of these comments came from the same organization (Integrated DNA Technologies).

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Codon Digest: Ghana Approves A Better Malaria Vaccine

Codon

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain.

Vaccine 52
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Codon Digest: Ghana Approves A Better Malaria Vaccine

Codon

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain.

Vaccine 52