香港六合彩中特网

A pioneering treatment, which allows people with haemophilia to produce the vital blood-clotting protein they lack, has reached Phase III trials and will hopefully provide a cure.

Gene therapy听is a novel听treatment听approach听that shows promise听for听a range of inherited听disorders听including haemophilia听鈥� a听condition that occurs when a single protein involved in blood-clotting is missing, resulting in听uncontrolled bleeding听at the slightest injury.听

Haemophilia affects more than 1 in 3,300 people worldwide. In听resource-poor countries it often remains undiagnosed and can lead to premature death.听

Normally when a blood vessel听breaks,听small blood听components called听platelets听clump and听work with听a听series of thirteen听(XIII)听protein factors听to form a听clot听over the injury.听But in听people with听haemophilia听A,听factor VIII听is missing and in haemophilia B听factor听IX听is missing.听

To replace the missing proteins,听Professor Amit听Nathwani听(香港六合彩中特网 Cancer Institute), and colleague听in the institute鈥檚 Department of Haematology听Professor Edward Tuddenham听spent a decade developing听gene therapy treatments听in collaboration with听researchers听at St Jude's Children's Research Hospital in Tennessee, USA.听

The team听uses a听harmless virus to听deliver听corrected copies of the听defective听clotting factor听gene to cells in the body听in a single injection. The cells can听then听manufacture听the听correct听form听of the听protein听allowing听blood to clot.听

鈥�鈥淭his single-dose treatment replaces the need for multiple injections and dramatically reduces the cost of treatment.鈥澨�

鈥淭his听single-dose听treatment replaces the need for听multiple听injections听and听dramatically reduces听the cost of treatment,鈥澨齟xplains Professor听Nathwani.听

This听successful use of听gene therapy听for haemophilia A听follows a similar trial听in听people with听haemophilia B听a decade听ago, when听Professor听Nathwani鈥檚听team听replaced听the missing Factor听ten (IX)听protein听using a single administration of gene therapy.听The majority of听these patients听have听since听been free of听spontaneous听bleeding听episodes.听听

Trials of the听Haemophilia A gene therapy show听a听single dose听is enough to听stop uncontrolled bleeding in patients for more than听three听years without any long-term toxicity.听听

鈥淭hese 鈥榦ne-dose-and-done鈥� gene therapies听provide a听potentially life-changing treatment for people with haemophilia,鈥澨齟mphasises听Professor听Nathwani, who听has听founded a company called Freeline Therapeutics to听commercialise听the technology.听

鈥淭he trial results听provide听inspiration and hope for听treating听a range of听single-gene听disorders听that currently lack effective treatment,"听he adds.听