Dr. Shalem’s research focuses on translational target discovery for a range of neurodegenerative diseases. He combines technology development of large-scale CRISPR-based perturbation screens with application of such technology together with additional genomic approaches.
Dr. Maris investigates the molecular and genetic mechanisms contributing to the development and progression of neuroblastoma, a common childhood cancer. He also aims to develop new molecular diagnostic tests and less toxic, targeted therapies to treat relapsed or refractory neuroblastoma, including a major effort in immunotherapy discovery and development.
The human genome provides a precise, biological blueprint of life. To implement this blueprint correctly, the genome must be read with great precision, but it's impossible for this process to be completely error-free. Mistakes during transcription - random errors in how DNA sequences are copied for a gene to be expressed - can happen any time in any number of ways.
By Bryan A. Wolf, MD, PhD, Chief Scientific Officer and Director of the Research Institute
Every space flight mission requires a carefully planned trajectory, and similarly the national Cancer Moonshot initiative needed a scientific roadmap to reach its ambitions of accelerating the prevention, diagnosis, and treatment of cancer to make a decade of progress in the next five years. In September,
Researchers at The Children’s Hospital of Philadelphia reported their latest results from their studies of an investigational personalized cell therapy for a highly aggressive form of cancer, acute lymphoblastic leukemia (ALL).