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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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INOVIO Announces Publication of Phase 1 Data from its COVID-19 DNA Vaccine Candidate, INO-4800 in The Lancet’s EClinicalMedicine

The Pharma Data

” Dr. Stanley Plotkin , Professor Emeritus at The Wistar Institute, said, “INOVIO’s DNA vaccine appeared to be quite safe with few significant reactions but yet induced both antibody and T cell responses to SARS-CoV-2.” About INOVIO’s DNA Medicines Platform. mg and 2.0

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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. ” I’ve long thought that SARS-CoV-2, or an immediate viral predecessor, emerged years before 2019 – at least as far back as 2013. But I think about that time often, and the warning that it sounded.

Virus 98
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Antibody-drug conjugates payloads: then, now and next

Drug Target Review

2 In response, DNA-damaging agents that could target the entire cell cycle received renewed attention as ADC payloads. More recently, researchers have begun to look at conjugating immunomodulatory agents to antibodies to directly activate an immune response against the tumour. Cancers 11 , 394 (2019). & Ohi, R.)

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The Forgotten Pandemic

Codon

million people with TB went undetected in 2019, but these estimates are uncertain due to inaccuracies in testing. In 2019, physicians were unable to tailor effective antibiotic regimens for 39 percent of new TB cases because the assessments they relied on could not gauge which antibiotics the strains would be susceptible to.

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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.

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Revolutionary nanoparticles enable gene-editing in lungs

Drug Target Review

Gene-editing methods allow researchers to modify DNA, resulting in alterations in physical characteristics such as eye colour and susceptibility to diseases. To achieve this, scientists utilise various technologies that function similarly to scissors, precisely cutting the DNA at a particular location.

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