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Latest Gene therapy Stories

2012-07-30 13:45:09

A five-country international team, led by Casey Eye Institute Molecular Diagnostic laboratory, BGI and Zhejiang University School of Medicine First Affiliated Hospital identified the NMNAT1 mutations as a cause of Leber congenital amaurosis (LCA), one of the most common causes of inherited blindness in children. The latest study was published online in Nature Genetics, reporting the genetic characteristics underlying some LCA patients, and providing important evidences that support NMNAT1 as...

2012-07-30 12:53:15

Researchers from the Massachusetts Eye and Ear Infirmary, The Children's Hospital of Philadelphia, Loyola University Chicago Health Sciences Division and their collaborators have isolated an elusive human gene that causes a common form of Leber congenital amaurosis (LCA), a relatively rare but devastating form of early-onset blindness. The new LCA gene is called NMNAT1. Finding the specific gene mutated in patients with LCA is the first step towards developing sight-saving gene therapy....

2012-07-26 06:46:08

(Ivanhoe Newswire) -- The sound of a horn beeping at you while driving, the chirping crickets after a huge rain storm, and your favorite new One Direction song -- imagine going through life not being able to hear any of it. That is a reality for those living with hearing loss. Now, researchers are trying new things to turn up the volume for the deaf. A new gene therapy approach can reverse hearing loss caused by a genetic defect in a mouse model of congenital deafness. New findings present...

2012-07-26 01:05:02

A new gene therapy approach can reverse hearing loss caused by a genetic defect in a mouse model of congenital deafness, according to a preclinical study published by Cell Press in the July 26 issue of the journal Neuron. The findings present a promising therapeutic avenue for potentially treating individuals who are born deaf. "This is the first time that an inherited, genetic hearing loss has been successfully treated in laboratory mice, and as such represents an important milestone for...

2012-07-26 01:03:22

A novel approach to gene therapy that instructs a person's own cells to produce more of a natural disease-fighting protein could offer a solution to treating many genetic disorders. The method was used to achieve a 2- to 3-fold increase in production of a protein deficient in patients with Friedreich's ataxia, as described in an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com) The article is available free...

European Agency Recommends Gene Therapy Approval
2012-07-21 08:56:42

redOrbit Staff & Wire Reports - Your Universe Online A European Union agency for the evaluation of medicinal products is recommending the approval of a gene therapy treatment for a rare disease, which would be the first time a treatment of this kind has received the regulatory go-ahead for use in the West, according to various reports published Friday. A statement released by the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) officially...

2012-07-09 10:54:16

The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children's Hospital study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies. A group of untreatable...

2012-07-03 10:25:59

Drugs are first to topically deliver gene therapy via commercial moisturizers for skin disease treatment “Getting under your skin” takes on a brave new meaning thanks to Northwestern University research that could transform gene regulation. A team led by a physician-scientist and a chemist -- from the fields of dermatology and nanotechnology -- is the first to demonstrate the use of commercial moisturizers to deliver gene regulation technology that has great potential for...


Word of the Day
conjunto
  • A style of popular dance music originating along the border between Texas and Mexico, characterized by the use of accordion, drums, and 12-string bass guitar and traditionally based on polka, waltz, and bolero rhythms.
The word 'conjunto' comes through Spanish, from Latin coniūnctus, past participle of coniungere, to join together; see conjoin