Published on in CHOP News
Results from multicenter study are first to show improved production of coagulation factor VIII over prolonged period, leading to reduction or complete elimination of bleeding events.
Published on in CHOP News
CHOP researchers have developed a novel cancer therapy that targets proteins inside cancer cells that are essential for tumor growth and survival but have been historically impossible to reach.
Published on in CHOP News
A collaborative team of researchers, including Dr. Stephan Grupp, recently presented preliminary data showing that a CRISPR-based gene-editing therapy for inherited blood disorders is safe and effective.
Published on in CHOP News
CHOP researchers have developed a “dimmer switch” system based on alternative RNA splicing that can control levels of proteins expressed from gene therapy vectors.
Published on in CHOP News
In a proof-of-concept study, CHOP researchers used a vector to edit a single base mutation in Hurler Syndrome, halting progression of a disease that causes irreversible damage before birth.
Published on in CHOP News
CHOP researchers were interviewed as a part of a comprehensive article about cell and gene therapy in Philadelphia Magazine.
Published on in CHOP News
In a proof-of-concept study, CHOP and Penn researchers have identified ionizable lipid nanoparticles that could be used to deliver mRNA as part of fetal therapy.
Published on in CHOP News
CHOP researchers identified an in vivo role for activated protein C (APC), which could potentially be exploited for future hemophilia A treatments.
Published on in In Utero Insights
Prenatal correction of cleft lip and/or palate would offer benefits that are not attainable through conventional postnatal treatment. Learn about our work to correct it in the laboratory.
Published on in CHOP News
The National Academy of Medicine elects two global leaders in cellular and gene therapy from Children’s Hospital of Philadelphia.