"Super pill" expected to be available within three years to treat more than 2,000 diseases

U.Sresearchers say they are developing a breakthrough new drug that could treat more than 2,000 geneticdiseases, including Duchenne muscular dystrophy and several types of cystic fibrosis
If clinical trials go well, the drug is expected to be available within three yearsThe results were published April 23 in the British journal NatureDuxing muscular dystrophy occurs in 1 in 3,500 men, a sexually linked recessive genetic disorder and the most common muscular dystrophy inchildrenchildren with the disease, may have trouble walking before school, between the ages of 7 and 12 years, almost all patients will lose the ability to walkIn their 20s, they all died ofheartand lung muscle failureDuxing muscular dystrophy is mainly due to the patient's muscle cells do not normally produce a protein called anti-myotrophin, resulting in muscle weakness throughout the patientBecause it is a sexual lysy recessive inheritance, onlymalewill have the conditionWomen who usually have an abnormal gene does not develop the condition, but have a 50% chance of inheriting the mutated gene on their son The genetic information stored in the human body's DNA is composed of four genetic codes: adenine (A), ostrich (G), thymus (T), and cytosine (C) After RNA transcription and splicing, thymus (T) is replaced by urinary (U) When RNA translates proteins, it forms a gene reading box with three genetic codes If the genetic code is missing or duplicated, it will cause the reading box downstream of the missing segment to be completely disrupted and useless proteins synthesized If the genetic code is not missing, but a genetic code changes to form a UAA, UAG, and UGA stop code, will also make the process of translation of proteins prematurely stopped Scientists speculate that a variety of human genetic diseases, including Duchenne muscular dystrophy, caused by genetic ally-righteous mutations The team, led by Dr Sweeney of the University of Pennsylvania, selected a new drug called PTC-124, which works in a similar way to gingantimy, which prevents ribosomes from identifying incorrect stop codes and continues to synthesize functional proteins, but unlike Gingdamin, PTC-124 does not affect normal stop codes, so safety is greatly improved Experiments in mice have shown that the drug has the effect of increasing the level of anti-myotrophin and restoring muscle function Dr Sweeney said the new treatment could help treat of a variety of genetic
diseases caused by genetically unrighteous mutations Maritta, director of the Muscular Dystrophy Society, said the findings were encouraging In the long run, the drug is expected to become a treatment for Duchenne muscular dystrophy (reprinted from Science and Technology Daily)