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genetics, disease & medicine

FDA Approves Gene Therapy for Spinal Muscular Atrophy
Ashley Yeager | May 27, 2019 | 3 min read
At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.
Allele Shows Pyrethroid Resistance’s Spread in African Mosquitos
Carolyn Wilke | Mar 22, 2019 | 4 min read
Researchers can now track the expansion of a resistance mechanism that allows the malaria vector Anopholes funestus to detoxify a key insecticide used on bed nets.
Lack of Diversity in Genetic Datasets is Risky for Treating Disease
Ashley Yeager | Mar 21, 2019 | 6 min read
Certain populations have been historically underrepresented in genome sequencing studies, but the NIH, private clinics, and 23andMe and other companies are trying to fix that.
Gene Drive–Equipped Mosquitoes Released into Lab Environment
Jef Akst | Feb 20, 2019 | 2 min read
The large-scale experiments aim to test how the technology would fare in the wild, if deployed to knock down populations of the pests.
AI App Identifies Rare Genetic Disorders from Photos of Patients’ Faces
Catherine Offord | Jan 8, 2019 | 2 min read
Deep-learning algorithms could help doctors narrow in on the causes of certain medical conditions, say researchers.
Babies’ Genomes Identify Risks Overlooked by Newborn Screens
Abby Olena, PhD | Jan 3, 2019 | 4 min read
A trial called BabySeq, in which researchers performed genomic sequencing on 159 newborns, identified children susceptible to diseases that regular screening doesn’t look for.
Claim of First Gene-Edited Babies Triggers Investigation
Catherine Offord | Nov 26, 2018 | 2 min read
He Jiankui says he has successfully used CRISPR-Cas9 to tweak the genes of twins born earlier this month. Some independent experts are dubious.
CRISPR Editing Heads Off Disease in Mouse Livers
Shawna Williams | Oct 9, 2018 | 2 min read
Separate proof-of-concept studies report success against two inherited diseases.
In Their Earliest Days, Embryos Record Their Environments
Kerry Grens | Oct 1, 2018 | 4 min read
Methylation patterns at so-called metastable epialleles in the genome stamp a memory into each of our cells.
Alzheimer’s Gene Affects Energy Consumption in Mouse Brains
Sukanya Charuchandra | Oct 1, 2018 | 2 min read
One variant limited the brain’s ability to use glucose.
US Companies Launch CRISPR Clinical Trial
Catherine Offord | Sep 3, 2018 | 2 min read
The Germany-based study will test an ex vivo genome-editing therapy for the inherited blood disorder β-thalassemia.
CRISPR Treatment for Duchenne Muscular Dystrophy Helps Dogs
Catherine Offord | Aug 31, 2018 | 2 min read
Researchers boosted levels of the dystrophin protein to nearly normal levels in a canine model of the disease.
Electric Shock Allows for CRISPR Gene Editing Without a Viral Vector
Ashley Yeager | Jul 12, 2018 | 2 min read
Brief electroporation appears to make T cells more receptive to new genetic material, which could speed the development of immunotherapies.
In Utero Transplant in First Clinical Trial Successful
Jim Daley | May 30, 2018 | 2 min read
Doctors treated the fetus, who has alpha thalassemia major, with cells from her mother’s bone marrow.
Placental Health Influences Baby’s Future Schizophrenia Risk, Study Suggests
Shawna Williams | May 29, 2018 | 1 min read
Complications during pregnancy may act via the placenta to magnify the effects of genetic risk factors.
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