Sentences with phrase «disorder duchenne»

A team from Duke University has used the gene editing tool — hailed as Science magazine's 2015 Breakthrough of the Year — for the first time to treat the genetic disorder Duchenne muscular dystrophy inside a living mammal, to be potentially replicated in humans.

That appears to be the case with Marathon Pharmaceuticals» deflazacort, a steroid that has now achieved FDA approval for treating the devastating muscle - wasting disorder Duchenne muscular dystrophy (DMD).

The saga of Sarepta Therapeutics» rare disease drug Exondys 51, the first - ever treatment for the degenerative movement disorder Duchenne muscular dystrophy, has taken yet another turn.

And Crispr - Cas9 isn't even the only type of Crispr out there: On April 12, researchers at the University of Texas Southwestern Medical Center announced they had successfully paired the gene - editing tool with a different kind of enzyme, called Cpf1, to correct mutations associated with the devastating muscle - wasting disorder Duchenne muscular dystrophy.

Not exact matches

These are the muscular dystrophies (among which are Duchenne and Becker); motor neuron diseases (including ALS and SMA); the peripheral nerve disorders (CMT and Friedreich's ataxia); inflammatory myopathies; disorders of the neuromuscular junction; metabolic diseases of muscle as well as other myopathies.

CRISPR and similar techniques for tweaking the genome may offer powerful ways to treat genetic disorders like Duchenne muscular dystrophy.

If you took high school biology in the 1990s, you probably learned about the molecular basis for human genetic disorders such as cystic fibrosis (1989), Huntingtons (1993), Duchenne and Becker muscular dystrophy (1987), and a rapidly growing list of single - gene disorders, and the correspondingly rapid growth in clinical diagnostic technology based on DNA sequence information, enabling certain diagnosis, sometimes before the advent of overt symptoms.

Duchenne Muscular Dystrophy (DMD) is a degenerative genetic disorder that affects the muscles in the body in a progressive manner, leading to eventual death.

Muscular dystrophies are an inherited group of disorders, and include Duchenne and Becker, Emery Driefuss, limb - girdle, myotonic, facioscapulohumeral, and distal types.

Albinism, Arthrogryposis Renal Dysfunction Cholestasis Syndrome, Autosomal Recessive Nonsyndromic Congenital Nuclear Cataract, Bare Lymphocyte Syndrome 2, Breast Cancer, Cholestasis, Progressive Familial Intrahepatic 1, Cholestasis, Progressive Familial Intrahepatic 2, Cholestasis, Progressive Familial Intrahepatic 3, Congenital Chloride Diarrhea, Congenital Muscular Dystrophy, Congenital Nephrotic Syndrome Finnish Type, Down syndrome, Duchenne muscular dystrophy, Epidermolysis bullosa, Hypoparathyroidism retardation dysmorphism syndrome, Joubert syndrome, Muscular dystrophy, Neurogenetic and neurometabolic disorders, Osteogenesis imperfecta, Sickle cell anemia, Spinal muscular atrophy, Thalassemia, Adolescent nephronophthisis, Infantile nephronophthisis

The authors were able to correct mutations in several well - characterized genetic disorders, including: Duchenne Muscular Dystrophy, Achondroplasia, and MECP2 - duplication syndrome using cells derived from human patients.

The genetic disease targeted is Duchenne muscular dystrophy (DMD), an X-linked recessive disorder affecting approximately 1 in 5000 males.