$26M grant to fund newborn genetic disease research
- National Institutes of Health will target disease using CRISPR tech
- The research will focus on finding treatment for PKU, HT1 and MPSI
- Doctor: "We will have data we need to move into clinical trials”
Latin American nurse monitoring on a premature newborn vital signs in an incubator while wearing a facemask during the COVID-19 pandemic
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(NewsNation) — With the help of a $26 million grant, a team of Philadelphia doctors will start treatment research for incurable genetic diseases that impact newborns.
The team from Penn Medicine and Children’s Hospital of Philadelphia will focus on Phenylketonuria (PKU), hereditary tyrosinemia type 1 (HT1), and mucopolysaccharidosis type 1 (MPSI), commonly known as Hurler’s Syndrome. The $26 million grant is funded by the National Institutes of Health.
A late diagnosis of PKU can lead to severe intellectual disability, seizures and psychiatric issues. HT1 is a genetic disorder, and if it’s untreated, it can lead to serious health problems. A newborn is diagnosed with Hurler’s Syndrome when the body doesn’t have enough of an enzyme to break down sugar molecules, which can cause numerous health problems including vision loss and impaired growth.
Researchers will utilize a technology called CRISPR to change single components in existing drugs to target different diseases.
“CRISPR offers the potential to develop highly effective treatments for incurable genetic diseases and improve the quality of life for patients with these conditions,” said William Peranteau, MD. “This NIH funding will help us develop and validate the safety of new gene therapies that can be given to patients in vivo, directly in the body, so that we will have the data we need to be able to move into clinical trials.”
