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Eclectic Genomics: Cat Flu, Dolphin Adaptation to Climate Change, Predicting Cancer, and Diagnosing Rare Disease

PLOS: DNA Science

Genomics applies to all species, revealing evolution in action, because we all use the same genetic code – that is, the correspondence between DNA sequences and the amino acid sequences of proteins. Cats and Bird Flu Comparing DNA sequences is a little like linguistic research that connects languages.

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Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

PLOS: DNA Science

million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it. The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. million, a lot less than the MLD treatment.

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Medicinal Chemistry In The Age Of Artificial Intelligence

LifeSciVC

In 2001, I was at Millennium Pharmaceuticals, one of the major players in the nascent field of genomics drug discovery, and that time has strong parallels to the changing state of drug development today. In fact, the Millennium of 2001 (pre-Takeda acquisition) had one drug approved which was Velcade (bortezomib).

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AI Tool CHIEF Paints a Landscape of a Cancer, Refining Diagnosis, Treatment, and Prognosis

PLOS: DNA Science

The most groundbreaking cancer drug approval was for Gleevec , with a mere 3-month trajectory at FDA before its approval in 2001. Diagnosis began to shift from a body-part basis to a molecular one, which I wrote about in “Mutation and location important in cancer treatment” for The Lancet in 2015.

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Defense-Forward Biosecurity

Codon

Allison Berke makes the case for real-time DNA sequencing and AI tools to detect pathogens before they spread widely. Reading DNA The first step in detecting a novel pathogen is recognizing it as an anomaly amidst a noisy background of other material. After copying the DNA to form a big pool, each piece is sequenced.

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Intellia Aims Big with CRISPR Therapy to Cure ATTR

The Pharma Data

Previous treatments could only hope to slow progression. Intellia has received approval to launch their Phase I trial of NTLA-2001 in hereditary ATTR. Their NTLA-2001 candidate uses their proprietary non-viral lipid nanoparticle platform, which will deliver this CRISPR/Cas9-based therapy.

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Genetic Signature for Drug Addiction Revealed in New Analysis of More Than A Million Genomes

PLOS: DNA Science

received treatment, according to the National Institute on Drug Abuse (NIDA). So scrutinizing genomes for genes that control drug dependency could inspire treatments. “RFLPs” are sites in a DNA sequence where a bacterial enzyme, called a restriction enzyme, cuts. Drug addiction is prevalent and deadly.

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