Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease and Lou Gehrig’s disease, is a rare condition characterized by the degeneration of nerve cells that control voluntary muscles. The disease leads to gradually worsening symptoms that include muscle weakness, twitching, and stiffness. As more of these nerve cells, or motor neurons, are lost, muscles decrease in size
Further action needed to facilitate development of safe, effective treatments Source The Pew Over the last two decades, cell therapies (which involve the transplantation of whole cells into a patient), gene therapies (which use genetic material to manipulate a patient’s cells), and other medical treatments intended to repair or replace damaged, diseased, or dysfunctional cells,
NIH Director Francis Collins describes evolution of existing collaborations and how the two agencies might facilitate development of gene therapy, such as by having a common template for regulatory review. Source Pink Sheet The National Institutes of Health is looking to build on its partnerships with the US Food and Drug Administration to tackle new
Discoveries Could One Day Lead to New Drugs to Prolong Healthy Human Lifespan The discovery in the 1990s that a mutation in a single gene of an experimental worm could double its lifespan set off a stampede of research on the molecular biology of aging and triggered hopes that drug therapies or other interventions could
Researchers have discovered that a key cell type involved in liver injury and cancer consists of two cellular families with different origins and functions. The research by academics from the Universities of Edinburgh and Bristol and funded by the Wellcome Trust and Medical Research Council, is published today [Tuesday 15 October] in Nature Communications. The distinguishing
A California “human biohacking” bill calls for warnings on do-it-yourself genetic-engineering kits. Source Technology Review It’s going to be illegal in California to sell “gene-therapy kits” unless they carry a warning that says not to use them on yourself. Just one wrinkle: we’re not sure any such kit exists. Not yet, anyway. The consumer protection
The process could be harnessed as a treatment for osteoarthritis Source Duke Health Contrary to popular belief, cartilage in human joints can repair itself through a process similar to that used by creatures such as salamanders and zebrafish to regenerate limbs, researchers at Duke Health found. Publishing online Oct. 9 in the journal Science Advances,
Researchers rapidly corrected finding through discussions on social media and preprints. Source Nature A study that raised questions over the future health of the world’s first gene-edited babies has been retracted because of key errors that undermined its conclusion. The research, published in June 2019 in Nature Medicine1, had suggested that people with two copies of a natural genetic mutation
Neurologic scores stabilized, seizures diminished with tailored antisense oligonucleotide therapy The researchers who developed a personalized antisense oligonucleotide (ASO) for a little girl with a form of Batten disease — all in record time — have detailed their case in the New England Journal of Medicine. Timothy Yu, MD, PhD, of Boston Children’s Hospital, and colleagues explained how
Subretinal administration of the gene therapy AAV2/5-OPTIRPE65 showed improvements in vision-guided mobility, retinal sensitivity, and foveal-driven visual function for patients with RPE65-associated retinal dystrophy, according to findings from a phase 1/2 study presented at the 2019 American Academy of Ophthalmology Annual Meeting. In the early-stage study, there was a significant change in the ability to complete a
