Children’s Hospital of Philadelphia Awards Gold Medal to Dr. Katherine High, Gene Therapy Pioneer

In an honor reserved for only the most significant achievements in advancing children’s health, Children’s Hospital of Philadelphia (CHOP) has awarded its Gold Medal to Katherine High, MD, a gene therapy pioneer and one of the lead developers of the first in vivo gene therapy approved by the Food and Drug Administration (FDA). Bestowed only 12 times in CHOP’s 167-year history, the Gold Medal highlights Dr. High’s groundbreaking discoveries at CHOP, which led to a gene therapy treatment for a rare form of inherited blindness and advanced gene therapy for hemophilia to late-stage testing.

Cardiologists, Cardiothoracic Surgeons Available for Expert Commentary Ahead of Upcoming Conferences

Cardiologists and cardiothoracic surgeons from the Smidt Heart Institute at Cedars-Sinai are available to discuss the latest advances in research, clinical care, transcatheter procedures and cardiothoracic surgery throughout the International Society for Heart and Lung Transplantation (ISHLT) Scientific Sessions 2022 and Heart Rhythm 2022.

UCI receives 5-year, $5 million CIRM award for training of diverse researchers

The University of California, Irvine has received a five-year, $5 million award from the California Institute for Regenerative Medicine to support a comprehensive doctoral, postdoctoral and clinical researcher training program to prepare the current and next generation of leaders in stem cell biology, gene therapy and regenerative medicine.

Children’s Hospital of Philadelphia Researchers Develop “Dimmer Switch” to Help Control Gene Therapy

In a major advancement in the field of gene therapy for rare and devastating diseases, researchers at Children’s Hospital of Philadelphia (CHOP) have developed a “dimmer switch” system that can control levels of proteins expressed from gene therapy vectors. The system is based on alternative RNA splicing using an orally available small molecule and works effectively in tissues throughout the body, including the brain. The first research regarding this innovation was published today in the journal Nature.

Mount Sinai researchers develop novel therapy that could be effective in many cancers

New York, NY (July 23, 2021) — Mount Sinai researchers have developed a therapeutic agent that shows high effectiveness in vitro at disrupting a biological pathway that helps cancer survive, according to a paper published in Cancer Discovery , a…

CHOP Researchers Use Base Editing in Preclinical Model to Correct Lethal Lysosomal Storage Disease Before Birth

Adding to the growing body of literature demonstrating the feasibility of correcting lethal genetic diseases before birth, researchers at Children’s Hospital of Philadelphia (CHOP) have used DNA base editing in a prenatal mouse model to correct a lysosomal storage disease known as Hurler syndrome. Using an adenine base editor delivered in an adeno-associated viral vector, the researchers corrected the single base mutation responsible for the condition, which begins before birth and affects multiple organs, with the potential to cause death in childhood if untreated.

Innovative Gene Therapy ‘Reprograms’ Cells To Reverse Neurological Deficiencies

A new method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study was led by researchers at The Ohio State University Wexner Medical Center and The Ohio State University College of Medicine.

New adaptable nanoparticle platform enables enhanced delivery of gene therapies

Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo. The work, led by researchers from RCSI University of Medicine…

Controlling insulin production with a smartwatch

Many modern fitness trackers and smartwatches feature integrated LEDs. The green light emitted, whether continuous or pulsed, penetrates the skin and can be used to measure the wearer’s heart rate during physical activity or while at rest. These watches have…

The ISSCR releases updated guidelines for stem cell research and clinical translation

Skokie, IL – The International Society for Stem Cell Research (ISSCR), today released updated guidelines for stem cell research and its translation to medicine. The update reflects emerging advances including, stem cell-based embryo models, human embryo research, chimeras, organoids, and…

Cholesterol levels sustainably lowered using base editing

Base editing is a novel gene editing approach that can precisely change individual building blocks in a DNA sequence. By installing such a point mutation in a specific gene, an international research team led by the University of Zurich has…

Penn Medicine and Children’s Hospital of Philadelphia to Host Symposium on the Future of Cell and Gene Therapies

Penn Medicine and Children’s Hospital of Philadelphia (CHOP) will host a virtual event on May 6 and 7 that will bring together cell and gene therapy leaders from the two institutions and around the world to discuss the latest achievements in the field, novel strategies, and future developments and applications for chimeric antigen receptor, CAR, T cell therapy and more.