My best friend's cousin is twelve and has Muscular dystrophy - he has been estimated another 6 years to live; is there any chance this would be available in time to cure him?
Thanks in advance x
First I am very sorry about your best friend's cousin. I wish the kid and his family as well as your friend the best.
It is true that they are developing Gene Therapy and some success have been observed in animals with genetically similar diseases. However, these animals were put on immunosuppresants as well. The transition from animals to humans can take quite sometimes, and before a therapy can be widespreadly used to treat Muscular Dystrophy ( I am guessing its Duchene Muscular dystrophy ) , it has to go through 4 stages of trials which can last up to 5 to 10 years.
The greatest problem in using gene therapy is that delivering the " corrected " gene to every muscle cells in the body is associated with greatly increased risk ( almost guaranteed) of provoking the host immune system to reject the therapeutic gene , thus making it difficult.
I would suggest that the boy's family should ask the hospital if there is any clinical trial currently running on the disease , and ask if the boy can be participated in the trial. I dont think there are many institutions that are running gene therapy trial in humans ( yet) , but its worthy to ask.
Sorry I am not much help. Again, I am truely sorry and wish the best of luck for the boy and his family. I really do hope the advanced therapy would soon become available.
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