A novel approach to treating a genetic condition that causes muscle weakness could soon be on the horizon , after a cistron - redaction proficiency called CRISPR was successfully used to alleviateDuchenne muscular dystrophy(DMD ) in computer mouse .
DMD is cause by a genetic mutation that inhibits the production of a protein address dystrophin . This vital protein helps to connect muscle fibers to the membranes that confine them , and is , therefore , essential for ensuring the structural unity of muscles . In its absence , muscles do not develop the right way , induce a mountain range of difficulty in developing children , often preventing them from walking . It can affect the chest and heart muscles , reducing a sufferer ’s life expectancy well .
The mutation that causes the condition is carried on the X chromosome , and , therefore , tend to affect boy far more commonly than girl . This is because males have only one tenner chromosome , while females have two , meaning that miss who inherit one faulty version of the gene for dystrophin are likely to also have one healthy reading , and can therefore still synthesize the protein .
Last week , three separate paperswere publish in the journalScience , each of which outlined an almost selfsame experimentation using CRISPR to treat DMD in mice . CRISPRinvolves introducing proteins , call in Cas9 proteins , into living cell . These protein then cut the DNA of those cells like genetic scissors , enabling mutations and other undesirable sequences – orexons – to be removed .
In this case , the researchers inserted the CRISPR - Cas9 complex into a virus which then infected the cells of mouse fetuses . They place fussy mutated exon on the cistron for dystrophin , which were responsible for for the mice ’s inability to produce the protein .
The squad noted that once these sport were tailor out of the mice ’s genomes , the animals were once again able to produce dystrophin , although sometimes this protein was slenderly cut short , since sure stretches of the desoxyribonucleic acid that encodes it were missing . Even in these sheath , however , the mice displayed a substantial improvement in symptoms , and did not suffer from DMD .
Though this is encouraging news , a number of stumbling block must be overcome before clinical trials on humans can begin . For example , it is currently not known how the computer virus used to give birth the CRISPR building complex will react with the human resistant system . For this ground , Eric Olson , who led one of the threestudies , told theNew York Timesthat “ we require to surmount up , improve efficacy and assess safety ” before any human studies can be launch .
