Biostrophin is a drug which may serve as a vehicle for

gene therapy Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DN ...

, in the treatment of Duchenne and

Becker Becker () is one of the German-language surnames, along with Bäcker and Baecker, that derive from the root, which refers to baking. The surname began as a name for a baker (and thus his family). In northern Germany it can also derive from the ...

muscular dystrophy Muscular dystrophies (MD) are a genetically and clinically heterogeneous group of rare neuromuscular diseases that cause progressive weakness and breakdown of skeletal muscles over time. The disorders differ as to which muscles are primarily affe ...

. As

mutation In biology, a mutation is an alteration in the nucleic acid sequence of the genome of an organism, virus, or extrachromosomal DNA. Viral genomes contain either DNA or RNA. Mutations result from errors during DNA or viral replication, mi ...

s in the

gene In biology, the word gene (from , ; "...Wilhelm Johannsen coined the word gene to describe the Mendelian units of heredity..." meaning ''generation'' or ''birth'' or ''gender'') can have several different meanings. The Mendelian gene is a ba ...

which codes for the protein

dystrophin Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane. This complex is variously known as the cost ...

is the underlying defect responsible for both disorders, biostrophin will deliver a genetically-engineered, functional copy of the gene at the

molecular A molecule is a group of two or more atoms held together by attractive forces known as chemical bonds; depending on context, the term may or may not include ions which satisfy this criterion. In quantum physics, organic chemistry, and bioche ...

level to affected muscle cells. Dosage, as well as a viable means for systemic release of the drug in patients, is currently being investigated with the use of both canine and

primate Primates are a diverse order of mammals. They are divided into the strepsirrhines, which include the lemurs, galagos, and lorisids, and the haplorhines, which include the tarsiers and the simians (monkeys and apes, the latter including huma ...

animal model An animal model (short for animal disease model) is a living, non-human, often genetic-engineered animal used during the research and investigation of human disease, for the purpose of better understanding the disease process without the risk of ha ...

s. Biostrophin is being manufactured by Asklepios BioPharmaceuticals, Inc., with funding provided by the

Muscular Dystrophy Association The Muscular Dystrophy Association (MDA) is an American 501(c)(3) umbrella organization that works to support people with neuromuscular diseases. Founded in 1950 by Paul Cohen, who lived with muscular dystrophy, it works to combat neuromuscular d ...

References

External links

Parent Project MD
Muscular dystrophy Genetic engineering {{Musculoskeletal-drug-stub

See also

Other drugs for Duchenne muscular dystrophy

References

External links