Genetic disorders are all caused by errors in the genetic code. People diagnosed with Down syndrome, Hemophilia, sickle cell anemia, muscular dystrophy and many other genetic disorders may someday be cured by ultrasound and gene therapy.
From Genetic Engineering Biotechnology News: The national institutes of health (NIH) awarded researchers at Allegheny General Hospital (AGH) a federal grant for over one million dollars to progress their development of a non-viral gene therapy approach that uses ultrasound to deliver DNA directly to target cells. The funds will support the use of ultrasound-assisted gene transfer in animals as a treatment for xerostomia, a complication of radiation therapy for head and neck cancer resulting from irreparable damage to salivary glands, which causes dry mouth and speech, chewing, and swallowing problems.
From TRIB Live News: Researchers at Allegheny General Hospital hope a $1.7 million federal grant will help develop new gene therapies to treat a salivary gland problem that affects millions of people. A team led by Michael Passineau, director of the Gene Therapy Program at the hospital’s Allegheny Singer Research Institute, will use the four-year grant from the National Institutes of Health to transfer therapeutic DNA into pig cells using ultrasound. Usually, the transfer is conducted using a virus.
From the New York Times: After more than two decades of dashed expectations, the field of gene therapy appears close to reaching a milestone: a regulatory approval. UniQure, a Dutch company, is headed by Jorn Aldag. The European Medicines Agency has recommended approval of a gene therapy to treat a rare genetic disease, according to the agency’s Web site. If the European Commission follows the advice, as it usually does, this would be the first regulatory approval of a gene therapy drug in the Western world. That could give a boost to the field, which at times has struggled for credibility and financing.
From BBC News: Just one injection could be enough to cure people with haemophilia B; they would no longer need medication, according to an early study in the UK and the US. Doctors believe the gene therapy will be "potentially life-changing". Other researchers have described it as a "truly a landmark study."
In the future, gene therapies will be used to replace misplaced, miscopied, and missing DNA with healthy genetic codes. This research will cure many patients born with genetic defects. In the past, however, many efforts have either failed to successfully cure patients or have seriously damaged a few test subjects. Without gene therapy cures, patients can only focus on managing and treating their genetic disorders. For example, people suffering from celiac disease treat themselves by removing all gluten from their daily diets; persons suffering from hemophilia receive artificial clotting chemicals, while preventing themselves from serious physical injury. Nevertheless, even with careful treatment of their genetic disorders, many people with these genetic diseases surrender to lifelong disability and physical death. A few science researchers believe that ultrasound and advanced gene therapy will be the new technique for delivering healthy genes directly to target cells to cure many people suffering from painful and debilitating genetic disorders.