Gene editing may prevent genetic and inherited diseases

For the first time, researchers have corrected a disease-causing mutation in early stage human embryos with gene editing. The technique, which uses the CRISPR-Cas9 system, corrected the mutation for a heart condition at the earliest stage of embryonic development so that the defect would not be passed on to future generations. With advances in stem … Read more

US FDA bans 23andme direct to consumer genetic testing kit

FDA Orders Genetic Testing Firm 23andme to Stop Marketing DNA Analysis Service — Blocks Sale of a Company’s Mail-Order Gene Tests – The Food and Drug Administration, USA has ordered 23andme – the maker of a popular genetic-testing kit to stop sales of its heavily marketed product, saying the mail-order tests haven’t been proven effective and could dangerously mislead people about their health.

Rare genetic disorder linked to schizophrenia

Rare genetic disorder points to molecules that may play role in schizophrenia — Finding may suggest novel therapeutic target for treating disease – Scientists studying a rare genetic disorder have identified a molecular pathway that may play a role in schizophrenia. The findings may one day guide researchers to new treatment options for people with schizophrenia ? a devastating disease that affects approximately 1 percent of the world’s population.

Gene therapy may restore sense of smell

Can’t smell anything? This discovery may give you hope – Gene therapy in mice restores sense of smell, may also aid research into other diseases caused by cilia defects, U-M researchers say – Scientists have restored the sense of smell in mice through gene therapy for the first time — a hopeful sign for people who can’t smell anything from birth or lose it due to disease. The achievement in curing congenital anosmia — the medical term for lifelong inability to detect odors — may also aid research on other conditions that also stem from problems with the cilia.

2 new pancreatic enzyme products Ultresa and Viokace to aid food digestion

FDA approves two new pancreatic enzyme products to aid food digestion — Approvals to help ensure adequate supply of these medications – Two new pancreatic enzyme products used to help aid food digestion, Ultresa (pancrelipase) and Viokace (pancrelipase), were approved today by the U.S. Food and Drug Administration.

Migraine in mom may increase baby’s risk of colic

Moms with migraines twice as likely to have baby with colic — Study shows moms who suffer migraines more likely to have colicky babies – Mothers who experience migraine may be more likely to have a baby with colic than mothers without a history of migraine. Colic is defined as excessive crying in an otherwise healthy infant.

New Down syndrome treatment suggested by US researchers

New Down syndrome treatment suggested by Stanford/Packard study — Cognitive dysfunction reversed in mouse model of Down syndrome. – Findings from the Stanford University School of Medicine and Lucile Packard Children’s Hospital shed light on the neural basis of memory defects in Down syndrome and suggest a new strategy for treating the defects with medication.

Stem cell – gene therapy may cure human genetic diseases

Combined stem cell-gene therapy approach cures human genetic disease in vitro – A study led by researchers at the Salk Institute for Biological Studies, has catapulted the field of regenerative medicine significantly forward, proving in principle that a human genetic disease can be cured using a combination of gene therapy and induced pluripotent stem (iPS) cell technology.

Genetic disease recreated in lab

US researchers watched genetic diseases unfolding in the laboratory after finding a way to make large numbers of affected cells. – When neurons started dying in Clive Svendsen’s lab dishes, he couldn’t have been more pleased. The dying cells ? the same type lost in patients with the devastating neurological disease spinal muscular atrophy ? confirmed that the University of Wisconsin-Madison stem cell biologist had recreated the hallmarks of a genetic disorder in the lab, using stem cells derived from a patient.

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