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

PLOS: DNA Science

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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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.

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ViiV Healthcare Announces Analysis Showing no Antiretroviral Therapy Interruptions Due to COVID-19 Across its Clinical Development Programme for Investigational, Long-Acting Cabotegravir and Rilpivirine

The Pharma Data

In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.

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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. CRISPR also addresses another limitation in CAR T therapies related to the insufficient quantity and poor quality of autologous T cells.

Disease 246
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Driving Toward Nanopores

Codon

And unlike traditional DNA sequencers, which parse genetic material by breaking it up into fragments and interpreting it chunk-by-chunk, a nanopore device unspools a long strand of DNA and reads it all at once. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast.

DNA 73
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PREVYMIS™ in Preventing Cytomegalovirus Infection and Disease in Adults Undergoing Allogeneic HCT

The Pharma Data

These studies involved patients predominantly in the United States, Italy, and Japan and employed a range of timepoints for initiation of PREVYMIS post-transplant (0 – 42 days) and duration of therapy (79 – 191 days). CMV is a common virus that infects people of all ages. About PREVYMIS (letermovir).

Disease 52
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Codon: Notes on Progress #2

Codon

“The recombinant DNA breakthrough has provided us with a new and powerful approach to the questions that have intrigued and plagued man for centuries. The central dogma is often depicted as DNA→RNA→protein, but it’s much more: A biophysical marvel inside the smallest of vessels. Biology is a Burrito.

DNA 52