2015 Disease DNA Regulations 
NIH Director's Blog: Drug Development
JANUARY 5, 2016
But before diving into our first “new science” post of 2016, let’s take a quick look back at 2015 and some of its remarkable accomplishments. Four of 2015’s Top 10 featured developments directly benefited from NIH support—including Science’s “Breakthrough of the Year,” the CRISPR/Cas9 gene-editing technique.
Drug Target Review
SEPTEMBER 4, 2023
There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.
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Drug Discovery World
JULY 15, 2022
The remaining 8% of the genome was often referred to as the dark matter of the genome or sometimes even ‘Junk DNA’ 2. The sequencing technology relied on could only yield sequences of relatively short DNA fragments, so the assembly of sequences from highly repetitive regions was a huge challenge.
Drug Discovery World
MARCH 6, 2024
The prevalence of these diseases has grown substantially in recent decades due to an increasing global population, longer life expectancies and increasing morbidity with predisposing risk factors. This is comparatively easier to accomplish in these diseases because they are relatively monogenic.
Drug Target Review
AUGUST 9, 2023
Published on bit.bio’s website, this article authored by Dr Tony Oosterveen dives into the close relationship between FTD and ALS with a focus on mechanisms underlying the neuropathology of both diseases. ALS is the most common motor neuron disease and is estimated to affect more than 80,000 people worldwide.
PLOS: DNA Science
APRIL 4, 2024
A Compelling Need I was delighted to discover that the inspiration for Lume is a rare, recessive genetic disease, trimethylaminuria (TMAU), aka “fish odor syndrome.” If each ingredient is recognized as safe, then manufacturing complies with FDA regulations. So the oft-elicited “clinically proven” is meaningless.
Advarra
AUGUST 10, 2023
Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Now, FDA’s CBER is setting its sights on making ambitious strides toward tackling rare diseases.
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