Hope for Gus Foundation hikes to Mount Everest Base Camp on September 25 to raise funds for medical research
Because of significant research breakthroughs in CRISPR, a new technology has the potential to correct mutations that cause Duchenne muscular dystrophy (referred to as DMD or Duchenne). Tonya Dreher, director of the Hope for Gus Foundation (named after her son who has the disease) is directing the foundation’s support to this research through PPMD.
Duchenne is a terminal, muscle-wasting genetic disorder, affecting one in 3,500 boys born each year. By age 12, most boys with DMD are in wheelchairs. They rarely live past their mid-20s (currently the average lifespan is 27). Researchers at the University of Texas Southwestern Medical Center have successfully corrected DMD in human cells and in mice, offering new hope that there will be a human trial for the treatment in the near future.
“We took patient-derived cells that had the most common mutation responsible for Duchenne muscular dystrophy and we corrected them in vitro to restore production of the missing dystrophin protein in the cells. This work provides us with a promising new tool in the CRISPR toolbox,” said author Dr. Eric Olson, chairman of Molecular Biology, co-director of the UT Southwestern Wellstone Muscular Dystrophy Cooperative Research Center, and director of the Hamon Center for Regenerative Science and Medicine, according to a statement on the university’s website. The research appears in the journal Science Advances.
Due to the tremendous potential of this research, Dreher is directing all funds raised from Hope for Gus’s Everest to End Duchenne trek to Olson’s lab.
The upcoming trek will take place in September of this year. Dreher will be hiking Mount Everest for a second time with a group that includes relatives, friends and colleagues of those who know or have lost someone to Duchenne. Members of the trekking team raise money and get sponsorships as they undertake this difficult trek.
“This is an extremely exciting development and we will do everything we can to provide support and move this research forward. Our goal, of course is to help the thousands of children and young adults suffering from this disease,” said Dreher, who started Hope for Gus in 2010 after learning of Gus’ DMD diagnosis.
To be part of the 2017 trek, you must be 18 or older (unless accompanied by a parent or guardian) You must be reasonably fit, able to walk five to eight hours a day, and possess an adventuresome spirit and desire to make a difference. The 75 mile, 17-day journey begins in Kathmandu and includes an exploration of Nepalese culture, visits to traditional villages, Buddhist mountain monasteries and bazaars, and culminates with the arrival to Everest Base Camp, high in the Himalayas, more than 18,000 feet above sea level. Although a demanding journey not meant for the faint of heart, trekkers need not be elite athletes or experienced climbers to complete or enjoy the journey. Participants are required to raise a minimum of $1,000 and are also responsible for individual travel expenses.
To learn more about Hope for Gus or becoming a part of the 2017 Everest to End Duchenne trek email firstname.lastname@example.org. The sign-up period closes Friday, August 25. Sponsorship opportunities associated with the hike are also available for businesses and corporations.