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CCMT News & Announcements
Displaying announcements from the past three years.
1 - 77 of 77
Published on Jun 21, 2024 in Cornerstone Blog
The Clinical Vector Core created a viral vector that changed the life of a child diagnosed with AADC deficiency.
Published on Jun 5, 2024 in Press Releases
Researchers from Children’s Hospital of Philadelphia (CHOP), the University of Iowa and the Rochester School of Medicine and Dentistry were awarded a Collaborative Grant from Emily’s Entourage.
Published on May 15, 2024 in Cornerstone Blog
Many of the great minds at CHOP convened on April 30 and May 1 to share how their research is expanding our understanding of pediatric neuroscience.
Published on May 10, 2024 in Cornerstone Blog
This week, honors for innovative CHOP investigators, early results of gene editing for inherited blindness, plus ASGCT and PAS meeting highlights.
Published on Apr 26, 2024 in Cornerstone Blog
News highlights this week include the Biomedical Technician Training Program recognition event, new COVID-19 outcomes research, and more.
Published on Apr 10, 2024 in Cornerstone Blog
Evan Weber, PhD, and a research team from CHOP and Stanford Medicine discovered that the protein FOXO1 plays a key role in regulating CAR T cells’ longevity and effectiveness.
Published on Mar 18, 2024 in Cornerstone Blog
Eight women in STEM at CHOP share their experiences in the field, current work, and advice for aspiring scientists.
Published on Mar 15, 2024 in Cornerstone Blog
This week, CHOP announces two new Frontier Programs, and researchers define the origins of hematopoietic failure in Fanconi anemia.
Published on Nov 24, 2023 in Cornerstone Blog
In The News highlights CHOP at the AHA Scientific Sessions, the new AACAP president, a preterm birth study, and new research recognitions.
Published on Nov 17, 2023 in Cornerstone Blog
Kristen Navarro, PhD, discusses her definition of diversity in science, her research on rare diseases, and her cat, Caramel.
Published on Sep 1, 2023 in Cornerstone Blog
This week’s news roundup features three studies, Doris Duke Award winners, and a commitment to the CHOP Parkway Run & Walk.
Published on Aug 29, 2023 in Cornerstone Blog
The Clinical In Vivo Gene Therapy group supports CHOP’s continued leadership in the in vivo gene therapy space.
Published on Aug 15, 2023 in Press Releases
CHOP researchers have developed a versatile and low-cost technology for targeted sequencing of full-length RNA molecules.
Published on Mar 31, 2023 in Cornerstone Blog
In part two of our series celebrating Women’s History Month, meet more women at CHOP working in science, technology, engineering, math, and medicine fields.
Published on Feb 6, 2023 in Cornerstone Blog
Two scientists with unique skill sets are developing an alternative method for gene therapy delivery with support from a CGTC Seed Grant.
Published on Feb 3, 2023 in Cornerstone Blog
In this news roundup, we celebrate research tools new and old, from RNA sequencing to gene editing to the best tool of all: collaboration.
Published on Nov 11, 2022 in Cornerstone Blog
This week’s research news roundup includes accolades for our scientists and the spaces they work in.
Published on Sep 19, 2022 in Cornerstone Blog
CHOP physician-scientists discuss their CAR T-cell therapy research for childhood cancers in Where Discovery Leads.
Published on Jul 8, 2022 in Cornerstone Blog
This week’s In the News features ASGCT meeting highlights, our new Nephrology Chief, and more.
Published on May 13, 2022 in Press Releases
Prestigious award recognizes Dr. High’s contribution to improving children’s health through her gene therapy research.
Published on May 12, 2022 in Cornerstone Blog
CHOP recognized gene therapy pioneer, Katherine High, MD, with Children’s Hospital of Philadelphia’s Gold Medal Award.
Published on Mar 4, 2022 in Cornerstone Blog
Learn about novel findings and new funding from our Children’s Hospital of Philadelphia researchers.
Published on Nov 19, 2021 in Cornerstone Blog
Dr. Lindsey George and colleagues report safe and sustained expression following hemophilia A gene therapy.
Published on Nov 5, 2021 in Cornerstone Blog
The Clinical Vector Core, an award-winning, state-of-the-art, multi-product GMP manufacturing facility, supports the growing field of gene therapy.
Published on Oct 18, 2021 in Cornerstone Blog
Raymond G. Perelman Center for Cellular and Molecular Therapeutics team highlights that extended safety testing of AAV-based RNAi strategies is critical.
Published on Aug 12, 2021 in Cornerstone Blog
Meet this year’s CHOP-RISES scholars, who engaged in a unique hybrid curriculum involving hands-on work in our labs and virtual learning.
Published on Jul 26, 2021 in Cornerstone Blog
Children’s Hospital of Philadelphia scientists developed a tunable switch system to control levels of gene therapy expression.
Children’s Hospital of Philadelphia Researchers Develop “Dimmer Switch” to Help Control Gene Therapy
Published on Jun 28, 2021 in Press Releases
Delivery system fine tunes gene therapy expression levels and may pave the way for a new wave of gene therapies to treat rare and complex diseases.
Published on May 26, 2021 in Cornerstone Blog
The 17th Research Institute Scientific Symposium, held virtually on May 20, highlighted advances in cell and gene therapy.
Published on May 14, 2021 in Cornerstone Blog
This week’s research news features Ran Barzilay, PhD, and the results of the latest COVID-19 Resilience Project from the Lifespan Brain Institute.
Published on May 12, 2021 in Press Releases
Four CHOP gene therapy experts were interviewed as part of a comprehensive article in Philadelphia Magazine about cell and gene therapy.
Published on Apr 30, 2021 in Cornerstone Blog
This week’s news round-up highlights the CCMT’s state-of-the-art Clinical Manufacturing Facility
Published on Mar 10, 2021 in Cornerstone Blog
We feature seven more women in STEM at CHOP nominated by leaders of their Centers of Emphasis.
Published on Mar 8, 2021 in Cornerstone Blog
Nominated by their Centers of Emphasis, women in STEM share what drives them, their words of wisdom, and why they do what they do.
Published on Aug 19, 2020 in Cornerstone Blog
Researchers will study the mechanisms behind novel pediatric neurodegenerative disorders and assess their role in adult-onset conditions.
Published on Mar 25, 2020 in Cornerstone Blog
Researchers leading fields historically underrepresented by women share words of wisdom for the next generation pursuing careers in STEM.
Published on Mar 11, 2020 in Cornerstone Blog
With a new NIH Innovator Award, Ophir Shalem, PhD, is developing an improved genetic screening method that directly targets proteins.
Published on Jan 31, 2020 in Cornerstone Blog
MOMS2 study findings show benefits of prenatal surgery for spina bifida continue to childhood. This and more In the News.
Published on Dec 13, 2019 in Cornerstone Blog
Learn about developments in chromatin rebuilding, novel hemophilia treatment, linking heart and kidney disease, and genetic sequencing.
Published on Dec 4, 2019 in Announcements
POSTPONED: The annual CHOP Research Institute poster day and Scientific Symposium celebration will be May 20 and 21 at the University of Pennsylvania.
Published on Nov 13, 2019 in Cornerstone Blog
Beverly Davidson, PhD, director of the Center for Cellular and Molecular Therapeutics, is advancing work on a gene therapy for Alzheimer’s.
Published on Oct 21, 2019 in Press Releases
Beverly L. Davidson, PhD, director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics has been elected into the National Academy of Medicine (NAM).
Published on Oct 21, 2019 in Press Releases
The National Academy of Medicine elects two global leaders in cellular and gene therapy from Children’s Hospital of Philadelphia.
Published on Oct 21, 2019 in Cornerstone Blog
Join us in congratulating cell and gene therapy experts Beverly Davidson, PhD, and Stephan Grupp, MD, PhD, newly elected to the National Academy of Medicine.
Published on Oct 18, 2019 in Cornerstone Blog
This week, researchers elucidate mechanisms of mitochondrial DNA mutations, identify a new neurodevelopmental syndrome, and more.
Published on Oct 2, 2019 in Cornerstone Blog
If there’s an injury on the mountain, these EMTs-on-skis come to the rescue. Han van der Loo, PhD, volunteers with the National Ski Patrol.
Published on Sep 20, 2019 in Cornerstone Blog
In this week’s recap, our researchers share discoveries in driving safety, concussion recovery, schizophrenia treatment, and more.
Published on Aug 9, 2019 in Cornerstone Blog
Heat waves, drenching rainstorms, and vacation escapes can’t stop our investigators from their continued advancement of scientific discovery.
Published on May 29, 2019 in Cornerstone Blog
The 2019 Scientific Symposium focused on the advances CHOP researchers have made in the booming area of genomics and data science.
Published on Apr 19, 2019 in Cornerstone Blog
Whether they study helmets on the football field or hemophilia in a lab, our scientists at Children’s Hospital of Philadelphia Research Institute are always on the cutting-edge of their respective fields, as the latest roundup of research news shows.
Published on Feb 18, 2019 in Cornerstone Blog
Stefano Rivella, PhD, is developing a novel gene therapy that could help patients living with sickle cell disease and other debilitating red blood disorders.
Published on Dec 21, 2018 in Cornerstone Blog
“The problem with too much clotting is by far one of the most staggering medical issues in the Western world,” Dr. Krishnaswamy said. See what he and his colleagues are doing to combat this deadly disease.
Published on Nov 16, 2018 in Cornerstone Blog
In this week's roundup, CHOP researchers help parents avoid unnecessary ER visits, and share real-world implications of injury prevention work.
Published on Nov 6, 2018 in Cornerstone Blog
Meet two of the scientists behind the game-changing gene therapies created by the Research Institute's Clinical Vector Core.
Published on Oct 8, 2018 in Cornerstone Blog
An infographic and article look at the science behind gene therapy, challenges faced by the field, and the road ahead.
Published on Jul 2, 2018 in Press Releases
A scientist in Children's Hospital of Philadelphia's (CHOP) Department of Biomedical and Health Informatics (DBHi) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics developed a new software tool that rapidly extracts phenotype information from…
Published on May 1, 2018 in Cornerstone Blog
Researchers gain new insights about dysfunction along an important biological pathway that may impair multiple organ systems in Huntington's disease.
Published on Apr 24, 2018 in Press Releases
CHOP Researchers Describe Biological Functions Relevant to Potential Treatments
Published on Mar 15, 2018 in Cornerstone Blog
Meet four women in science who are driving discovery at Children's Hospital of Philadelphia Research Institute.
Published on Dec 21, 2017 in Cornerstone Blog
Landmark gene therapy approval opens door to develop therapies that target other mutations behind hereditary blindness and retinal diseases.
Published on Dec 19, 2017 in Press Releases
Children’s Hospital of Philadelphia (CHOP) today joins with other research leaders in celebrating the U.S. Food and Drug Administration’s (FDA) approval of Spark Therapeutics’ innovative gene therapy for the treatment of a rare, inherited form of retinal blindness.
Published on Sep 8, 2017 in Cornerstone Blog
September marks National Childhood Cancer Awareness Month, and this year at Children's Hospital of Philadelphia, we kick-started the commemorative period on the heels of exciting news about breakthroughs in pediatric cancer immunotherapy research.
Published on Aug 29, 2017 in Cornerstone Blog
For children with hemophilia, every new research advance is a step toward a life filled with more activity, freedom, and adventure. The genetic condition, which affects roughly one in 5,000 births, causes children to bleed and bruise more easily than others ‚Äì meaning that a…
Published on Apr 21, 2017 in Cornerstone Blog
A simple question raised by a concerned parent can often kick-start lines of research at Children's Hospital of Philadelphia. This week in the news, those sorts of everyday queries - whether they're about how to breastfeed, why parents should follow a vaccine schedule…
Published on Apr 18, 2017 in Press Releases
Neuroscientist at Children’s Hospital of Philadelphia Joins an Elite National Honorary Society
Published on Jul 29, 2016 in Cornerstone Blog
Welcome back to another weekly edition of our roundup of research news from The Children's Hospital of Philadelphia! One of the things that is so exciting about research at CHOP is that our researchers are working to improve the health and lives of children in such a broad…
Published on Jan 25, 2016 in Cornerstone Blog
Research at The Children’s Hospital of Philadelphia never stops. To help keep you informed about new discoveries and CHOP researchers’ views on timely topics, we are bringing together a roundup of news highlights. Look for this as a recurring feature here on Cornerstone.
Published on Dec 29, 2015 in Cornerstone Blog
Although the year is coming to a close, the research achievements at The Children’s Hospital of Philadelphia in 2015 remain enduring contributions to pediatric health.
Published on Nov 12, 2015 in Cornerstone Blog
When something important is missing, we often search for a replacement. After many years of looking, a team of researchers at The Children’s Hospital of Philadelphia and the University of Missouri have found a way to substitute for a missing gene linked to a relentless childhood…
Published on Apr 24, 2015 in Cornerstone Blog
Scientists at The Children’s Hospital of Philadelphia are comparing two molecular therapy techniques for Dystonia — RNA interference (RNAi) and antisense oligonucleotides (ASOs).
Published on Jan 21, 2015 in Cornerstone Blog
The Children’s Hospital of Philadelphia (CHOP) today announced a $50 million gift from Raymond G. Perelman.
Published on Jan 12, 2015 in Cornerstone Blog
Researchers in the Center for Cellular and Molecular Therapeutics (CCMT) at The Children’s Hospital of Philadelphia suggest that an intricate pathway crucial to the development of Huntington’s disease (HD) rests on a “biological teeter-totter” that when carefully balanced could…
Published on Apr 2, 2014 in Cornerstone Blog
Beverly L. Davidson, PhD, a nationally prominent expert in gene therapy, joined The Children’s Hospital of Philadelphia on April 1.
Published on Jan 16, 2014 in Cornerstone Blog
High is one of the world’s leading experts in gene therapy, which has long been a “next big thing” in medicine: Take a person with a devastating genetic disease and replace their nonfunctional gene with a normal one — a cure built right into your DNA.
Published on Dec 11, 2013 in Cornerstone Blog
For her pioneering hemophilia and gene therapy research, Children's Hospital hematologist Katherine A. High, MD, director of the Center for Cellular and Molecular Therapeutics (CCMT), was recently honored with the 2013 E. Donnall Thomas Lecture and Prize at the American…
Published on Jul 25, 2013 in Cornerstone Blog
Gene therapy researchers at The Children’s Hospital of Philadelphia have produced a bioengineered decoy that by fooling the immune system prevents it from undermining the benefits delivered by a corrective gene. The decoy could offer a new treatment for genetic diseases while…
Published on May 2, 2013 in Cornerstone Blog
A groundbreaking clinical trial of gene therapy for a form of congenital blindness, sponsored by The Children’s Hospital of Philadelphia in collaboration with Penn Medicine, was recently recognized with the Distinguished Clinical Research Achievement Award from the Clinical…