YOUR SUPPORT MATTERS.
Suneel’s Light was established with the sole mission to raise awareness while funding critical research for treatments in search of treatments and a cure for Duchenne Muscular Dystrophy. Much progress has been made in 2017, resulting in recently approved drugs by the FDA. In addition, our charitable foundation directly supports Western New York families affected by this devastating disease through special projects designed to improve the quality of their lives.
HERE'S HOW your support isMAKING A DIFFERENCE
Suneel’s Light Funds Exon Skipping Pioneer
DMD is a rare disease almost exclusively affecting young men. Due to a genetic defect, the body does not produce dystrophin, which ensures the proper function of muscle tissue. Exon skipping is a way to help make the genetic code readable again.
Exon skipping is a form of RNA splicing technology used to cause cells to “skip” over faulty or misaligned sections of genetic code, leading to a truncated shorter form of dystrophin that still works despite the genetic mutation.
Suneel’s Light is especially proud of the approval of Exondys 51, as our Foundation supported the research and early development of exon skipping technology through a grant to Dr. Steven Wilton of the University of Western Australia, a pioneer in exon skipping technology. The FDA approval brings hope to thousands afflicted with DMD throughout the world.
Research is at the heart of advances in treatment and care for Duchenne Muscular Dystrophy. Through your generous donations, the Suneel’s Light is proud to have played a role in supporting the most promising research in the development of these therapies. We will continue to support research and development, and provide strong advocacy to advance treatments for DMD. Let’s keep the drive alive towards finding that cure!
DMD Research Offering
Radical New Hope
Gene therapy, in which a replacement gene is delivered to a person’s cells, is offering radical new hopes for boys with DMD. The first muscular dystrophy gene therapy study is scheduled to begin soon at Nationwide Children’s Hospital in Columbus, Ohio.
The therapy involves “microdystrophin,” foreshortened copy of the dystrophin gene that’s small enough to fit inside a virus, which is required to shuttle the genetic information into a person’s cells. The full size dystrophin gene is just too big to fit into a benign virus. Although the mini-genes won’t be perfect, doctors hope that boys who receive them will end up with a mild handicap, instead of a death sentence.
The red-hot genome editing tool known as CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) has been used to treat a severe form of muscular dystrophy in mice. Three group of researchers used CRISPR to snip out part of a defective gene in mice DMD, allowing the animals to make an essential muscle protein, dystrophin. This approach is the first time CRISPR has been successfully delivered throughout the body to treat grown animals with a genetic disease.
The technology, dubbed “2015’s Breakthrough of the Year” by the journal Science, relies on a strand of RNA to guide an enzyme called Cas9 to a precise spot in the genome, where the enzyme snips the DNA. Cells then repair the gap either by rejoining the broken strands or by using a provided DNA template to create a new sequence. This exciting approach is ripe for clinical translation.
For more information, read the August 2017 article in MIT Technology Review.
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Please call Tom Maher at
716.243.0882 to find out how you can join our organization and make a difference in the lives of these amazing children.