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 D ...
, in the treatment of
Duchenne and
Becker 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 af ...
.
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, m ...
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 b ...
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 cos ...
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 bioch ...
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
Canine may refer to:
Zoology and anatomy
* a dog-like Canid animal in the subfamily Caninae
** '' Canis'', a genus including dogs, wolves, coyotes, and jackals
** Dog, the domestic dog
* Canine tooth, in mammalian oral anatomy
People with the ...
and primate
Primates are a diverse order (biology), order of mammals. They are divided into the Strepsirrhini, strepsirrhines, which include the lemurs, galagos, and lorisids, and the Hapl