My penn medicine careers11/17/2023 ![]() ![]() To validate this approach, a research team led by Laura Breda, PhD, and Michael P. One option that would eliminate the need for the above methods would be in vivo gene editing, in which gene editing tools are infused directly into the patient, allowing HSCs to be edited and corrected without the need for conditioning regimens. These conditioning procedures come with significant toxic side effects, underscoring the need to investigate less-toxic approaches. The former approach comes with the risk of graft versus host disease, given that the HSCs come from a donor, and both processes involve a conditioning regimen of chemotherapy or radiation to eliminate the patient's diseased HSCs and prepare them to receive the new cells. In patients with non-malignant hematopoietic disorders like sickle cell disease and immunodeficiency disorders, these blood cells don't function correctly because they carry a genetic mutation.įor these patients, there are currently two avenues for potentially curative treatments, both of which involve a bone marrow transplant: a stem cell transplant with HSCs from a healthy donor, or gene therapy in which the patient's own HSCs are modified outside of the body and transplanted back in (often referred to as ex vivo gene therapy). Hematopoietic stem cells (HSCs) reside in the bone marrow, where they divide throughout life to produce all cells within the blood and immune system. A targeted mRNA-encoded genomic editing methodology could lead to controlled expression, high editing efficacy, and potentially safer in vivo genomic modification compared to currently available technologies." Here, we combined the targeted platform with advances in mRNA therapeutics and RNA-based genomic editing tools to provide a new way of controlling hematopoietic stem cell fate and correcting genetic defects. "In our study, we are providing a cell-specific targeted lipid nanoparticle encapsulating mRNA therapeutics/editors as a platform technology that can be used for in vivo cellular reprogramming in many diseases in need of a precisely targeted gene therapy modality. "Targeted delivery of mRNA-encoded therapeutics to specific tissues and cell types will have an immense impact on the way diseases will be treated with nucleic acids in the future," said senior author Hamideh Parhiz, PharmD, PhD, a research assistant professor of Infectious Diseases at Penn. This is a big step forward in how we think about treating genetic diseases and could expand the access of gene therapies to patients who need them most." "In our paper, we have shown that it is possible to replace diseased blood cells with corrected ones directly within the body in a 'one-and-done' therapy, eliminating the need for myeloablative conditioning treatments and streamlining the delivery of these potentially life-changing treatments. ![]() "Right now, if you want to treat hematologic diseases like sickle cell disease and beta thalassemia with gene therapy, patients must receive conditioning treatments like chemotherapy to make space for the new, corrected blood cells, which is both expensive and comes with risks," said co-senior author Stefano Rivella, PhD, Kwame Ohene-Frempong Chair on Sickle Cell Anemia and Professor of Pediatrics at Children's Hospital of Philadelphia. News & World Report as one of the nation’s best hospitals in 2017-18.Ĭurrent employees please apply by clicking on your "career worklet" in Workday.This approach could expand access and reduce the cost of gene therapies for blood disorders. It has been recognized regionally and nationally for clinical excellence and patient safety and ranked nationally by U.S. Lancaster General Hospital has been designated a Magnet hospital for nursing excellence four times. Lancaster General Health Physicians is a network of more than 300 primary-care and specialty physicians, at more than 40 offices throughout the region. Outpatient services are provided at the Downtown and Suburban pavilions, along with additional outpatient facilities and Express and Urgent Care locations throughout the region. Our membership in Penn Medicine brings together the strengths of a world-renowned, not-for-profit academic medical center and a nationally recognized, not-for-profit community healthcare system. Lancaster General Health, a member of the University of Pennsylvania Health System (Penn Medicine), is a 719-licensed bed not-for-profit health system with a comprehensive network of care encompassing Lancaster General Hospital, Women & Babies Hospital and the Lancaster Rehabilitation Hospital (in partnership with Kindred Healthcare). Careers at Lancaster General Health (View Available Jobs) ![]()
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