Remove 2019 Remove DNA Remove Immune Response Remove Virus
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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. Affected children rarely live past infancy.

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AI in gene delivery vector discovery and design

Drug Target Review

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Nature reviews Drug discovery 18 , 358-378 (2019).

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Can Global Genomic Surveillance Forecast the Next Pandemic?

PLOS: DNA Science

Many of the links below are to some of the 100+ DNA Science posts that I wrote during the pandemic. A Novel Virus Appears In retrospect, everything unfolded with astonishing speed. ” I’ve long thought that SARS-CoV-2, or an immediate viral predecessor, emerged years before 2019 – at least as far back as 2013.

Virus 98
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HOOKIPA Announces Positive Phase 2 Interim Safety, Immunogenicity, and Efficacy Data for its Cytomegalovirus Vaccine Candidate HB-101

The Pharma Data

Compared to placebo, participants vaccinated with three HB-101 doses had: a 48 percent reduction in CMV viremia (presence of CMV DNA in the blood); a 42 percent reduction in the use of antiviral therapy; and. 3 The majority of CMV infections are not serious, and the virus can lay dormant in the body for years.

Vaccine 52
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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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RRx-001: Jack of all trades, master of…many

Drug Discovery World

The lead AdAPT candidate, AdAPT-001, in a Phase I/II clinical trial for the treatment of cancer, carries an extra piece of DNA called a transgene that binds to, traps, and neutralises a molecule called transforming growth factor-beta (TGF-b), which tumours hijack to suppress the antitumour immune response. Future Oncol.