Thaindian News

London, Mar 1 (ANI): Scientists at Ohio State Uni­vers­ity say that a lethal childhood disorder spinal muscular atrophy may be susceptible to effective treatment through gene therapy.

Re­search in mice showed that the ther­a­py could re­verse a pro­tein de­fi­cien­cy char­ac­ter­is­tic of the con­di­tion and thus cor­rect motor func­tion, re­store nerve sig­nals and im­prove sur­viv­al.The mice were en­gi­neered to de­vel­op the dis­or­der. The dis­ease re­sults when due to a mis­sing gene, mo­tor neu­rons - nerve cells that send sig­nals from the spi­nal cord to mus­cles - pro­duce too lit­tle of what is called sur­viv­al mo­tor neu­ron pro­tein.The re­search­ers used an al­tered vi­rus to de­liv­er a por­tion of DNA that makes the pro­tein in­to the veins of 1- to 10-day-old mice.

The vi­rus in­jected in­to the youngest mice reached al­most half of their mo­tor neu­rons, re­sult­ing in im­proved mus­cle co­ordina­t­ion, prop­erly work­ing elec­tri­cal sig­nals to the mus­cles and long­er sur­viv­al, sci­en­tists said.”When you put the pro­tein in post­na­tally, it will res­cue the ge­net­ic de­fect. This tech­nique cor­rects the mice con­sid­erably more than any drug cock­tails be­ing stud­ied as a po­ten­tial treat­ment in hu­mans,” said Ar­thur Burghes, bio­chem­ist at Ohio State Uni­vers­ity and a sen­ior co-author of the stu­dyGene ther­a­py is a rel­a­tively new class of ex­per­im­en­tal treat­ments in­volv­ing in­tro­duc­ing genes in­to pa­tients through var­i­ous means in or­der to re­place de­fec­tive genes or oth­er­wise help fight ill­ness.

Al­though gene ther­a­py has sparked great ex­cite­ment among sci­en­tists, the re­search has suf­fered set­backs, in­clud­ing a 1999 death dur­ing a clin­i­cal tri­al for or­ni­thine tran­scar­boxy­lase de­fi­cien­cy.

Oth­er stud­ies have shown better re­sults, but the U.S. Food and Drug Ad­min­istra­t­ion has to date not ap­proved any gene ther­a­py treat­ments.The sci­en­tists used a spe­cial form of a vi­rus to de­liv­er the pro­tein to nerve cells in the mice.

Bri­an Kas­par, an in­ves­ti­ga­tor in the Re­search In­sti­tute at Na­tion­wide Chil­dren’s Hos­pi­tal and as­sis­tant pro­fes­sor of pe­di­at­rics at Ohio State, al­so a sen­ior co-author of the stu­dy, said that this vi­rus still has the ca­pa­bil­ity to in­fect cells but has been al­tered so it will not copy it­self and cause ill­ness in hu­mans.

The study has been pub­lished on­line in the jour­nal Na­ture Bi­o­tech­nol­ogy. (ANI)