American Diabetes Association $1.625M Grant to Support Gene Editing for Type 1 Diabetes
2.2.16 – PR Newswire
The Program in Cell and Gene Therapy (PCGT) in the Center for Immunity and Immunotherapies has received a $1.625 million five-year Pathway Visionary Award from the American Diabetes Association. Funding will support the application of PCGT’s gene editing technology to find a way to prevent and reverse type 1 diabetes. The Pathway to Stop Diabetes project aims to edit genes in regulatory T cells in order to preserve their function and protect the beta cells from autoimmune attack, potentially preventing or reversing type 1 diabetes.
Seattle Children’s Researchers Pioneer Gene Editing That Kills, Resists HIV
9.30.15 – On the Pulse
Dr. David Rawlings and Dr. Andrew Scharenberg, researchers at the Center for Immunity and Immunotherapy at Seattle Children’s Research Institute, published a paper today in Science Translational Medicine that reveals a groundbreaking approach to engineering human T cells, which are crucial for fighting infection and show promise in treating autoimmune disorders, blood diseases and some types of cancer.
Seattle Children’s Research Institute Teams Up With bluebird bio to Pioneer Genome Editing and Gene Therapy Research in Pediatric Diseases
9.10.15 – Press Release
Gene therapy research aims to cure pediatric diseases early in life by targeting and repairing the disease-causing genes in a patient’s own genome.
‘Made a difference’: Stem cell donor meets her tiny recipient
7.27.15 – Hutch News
Canadian woman’s stem cells helped cure severe immune disorder in baby Savanna.
Seattle Children’s Aims to Partner with Medical Industry to Develop Pediatric Therapies
2.18.15 – On the Pulse
Seattle Children’s is hoping to bridge the gap between promising research and potential treatments and cures for pediatric diseases by partnering with drug manufacturers and biotech companies through its new Office of Science-Industry Partnerships.
The Cruelest Equation
The Chattanooga Times Free Press
“One in a million” was how doctors described the genetic disease called IPEX. Still, it found its way into two of her three sons. “Normal,” the boys’ newborn screening tests had said. Yet one hidden, molecular glitch had triggered their bodies to self-destruct. Dr. Troy Torgerson of Seattle Children’s is featured in this article.
Everyday Heroes: The Barrett Family
10.24.14 – KATU News (Portland, Oregon)
After the death of her baby boy, Stacey Barrett has been on a mission to keep other newborns from dying of severe combined immunodeficiency.
'It's a miracle': 3-year-old from Scituate battles rare disease
10.14.14 – The Patriot Ledger (Quincy, Mass.)
Three-year-old Anthony “Sonny” D’Ambrosio is a big boy with cherubic cheeks and a beaming smile. Sonny was born with the rare genetic condition immunodysregulation polyendocrinopathy enteropathy X-linked syndrome, known by the acronym IPEX. In a person with IPEX, T cells, which are important to the body’s immune system, work overtime and attack the body’s tissue and organs, Nicole D’Ambrosio said. The condition is passed from mothers to sons; girls are only rarely affected. Sonny “has plenty of T cells, but there’s just no way for his body to shut them off,” D’Ambrosio said. Sonny has been treated most of his life at Boston Children’s Hospital, but earlier this month his family moved from Scituate to Seattle to be closer to Dr. Troy Torgerson, an IPEX specialist at Seattle Children’s.
Simple test at birth saves baby from ravaging disease
10.3.14 – KOMO 4 News
Two babies in our state were born with an immune system disorder, within months of each other. One died after simply catching a cold. The other is thriving today, thanks to a simple difference. He was born after state-mandated testing for the disease. With early detection, doctors were able to save his life.
Transplant Saves First ‘Bubble Boy’ in Wash. State Detected with Newborn Screening
8.12.14 – On the Pulse
Ezra Dixon was born April 7, four months after the state of Washington first starting screening newborns for the disorder commonly known as “bubble boy disease,” which leaves its patients at the mercy of common germs.
More children, young adults being diagnosed with IPEX syndrome
5.4.14 – The Colorado Springs Gazette
Doctors have increasingly diagnosed children and young adults with an autoimmune disorder called IPEX syndrome. The reason dates to 2001, when CIIT researchers published a landmark paper describing the syndrome’s link to the FOXP3 gene.
5K race raises awareness of SCID, honors infant
10.3.13 – The Oregonian
The Friends of Jordan 5K Memorial Fun Run and Walk raised money to promote the need for screenings for severe combined immunodeficiency, or SCID, in Oregon, and to help fund screenings for at-risk families that can’t afford them. The race was launched in honor of Jordan Janeway, who died of SCID when he was 9 months old. He was treated by Dr. Suzanne Skoda-Smith, who sees patients in Seattle Children’s Immunology Clinic and conducts research at the CIIT.