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Research paper on gene therapy

research paper on gene therapy

work with this compound that continues to show promise. A second strategy to correct calcium accumulation in the cell is being developed by Akashi Therapeutics. For treatment of DMD, then, scientists hope that stem cells could someday be transplanted (from healthy donors) to grow new muscle. Most people feel that it is okay to use gene therapy to treat human genetic diseases. Additional research involves a virus known as adenovirus. Somatic therapy uses cells that are not naturally reproductive. Additional studies investigating the optimal drug regimen for slowing cardiac decline in DMD include a phase 4 study in Italy comparing the effects of carvedilol (a beta blocker) with Ramipril (an ACE inhibitor and a phase 3 study in France examining the effects of nebivolol. Should it be used at all? The drug DT-200 is an oral sarm in development by Akashi Therapeutics, and has shown positive effects in early studies.

MDA has supported research by a company called Tivorsan that has developed an engineered version of biglycan called TVN-102 that could be injected into patients. PB1046 is an engineered version of Vasoactive Intestinal Peptide (VIP a neuropeptide which has been shown to be ionotropic (increases contraction of the heart) and lusitropic (speeds relaxation of the heart). The drugs coax cells to ignore, or "read through a premature stop codon in a gene. Is developing a therapy based on angiotensin 1-7, called TXA127. Potential therapies that utilize muscle specific promoters are currently in phase 1 clinical testing for DMD (K.micro-Dystrophin) and also phase 1-2 testing for lgmd (CK. In addition, gene therapy may some day prove useful in the treatment and curing of such common disorders as cancer, aids, and the common cold. One strategy that has received considerable MDA support involves inhibiting the actions of a naturally apush explain essays occurring protein called myostatin that limits muscle growth. The key difficulties researchers are working to overcome include dealing with the large size of the dystrophin gene, delivering a sufficient quantity of the new genes to muscle (while avoiding other tissues and avoiding an unwanted immune response to the proteins made from the new.

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