PLOS: DNA Science

AUGUST 17, 2023

The Maui firestorm was so vast and fast that most identification of human remains will come from bits of persisting DNA from mitochondria. MtDNA in Forensics and Genealogy Mitochondrial DNA (mtDNA) is used in forensics to match the tiniest bits of human remains to families. Mitochondrial DNA is also far more abundant than nuclear DNA.

DNA 98

DNA Disease Science Research 98

PLOS: DNA Science

JULY 27, 2023

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.

Disease 98

Disease DNA Virus RNA 98

DrugBaron

JANUARY 8, 2021

Here is a disease that is caused by the lack of functional dystrophin in the muscles of these boys. The underlying assumption of most gene therapy procedures, irrespective of the disease being targeted, is that restoration of a wild-type DNA sequence will alone be sufficient to normalise the phenotype of the individual.

DNA 100

DNA Therapies Disease Production 100

PLOS: DNA Science

MARCH 21, 2024

Lenmeldy adds functional genes that encode the enzyme arylsulfatase A (ARSA) to bone marrow stem cells taken from children who have inherited the disease, but haven’t yet developed symptoms, which typically begin around age 2 or 3. Each of their children would face a 1 in 4 chance of inheriting the disease.

Therapies 98

Therapies Clinical Trials DNA Trials 98

LifeSciVC

AUGUST 5, 2024

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).

Molecular Biology 85

Molecular Biology Production Small Molecule Drug Development 85

Codon

AUGUST 27, 2024

Allison Berke makes the case for real-time DNA sequencing and AI tools to detect pathogens before they spread widely. Doctors in training are told that when they hear hoofbeats, they should think horses, not zebras; rare diseases are the exception, not the rule. After copying the DNA to form a big pool, each piece is sequenced.

DNA 83

DNA Laboratories Hospitals Virus 83

The Pharma Data

OCTOBER 19, 2020

Transthyretin Amyloidosis (ATTR) is a rare, progressive and fatal disease. 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.

Therapies 52

Therapies Pharmacokinetics Trials Clinical Trials 52