UROP Project

Gene therapy for the treatment of blood disorders



Daniel Holder

Program Director UROP


+49 241 80-90695


Key Info

Basic Information

Project Offer-Number:
UROP Abroad
Biotechnology / Molecular Biotechnology
Organisation unit:
University of Pennsylvania - Children's Hospital of Philadelphia
Language Skills:
Fluent English


We are a lab co-associated with the University of Pennsylvania, an Ivy League school, and The Children's Hospital of Philadelphia. Both are world-renowned institutions and pioneers in pediatric hematology research. Children from around the world seek treatment at CHOP, and countless more are affected by research both at CHOP and Penn. Our laboratory has a strong translational focus, with two main areas: • gene therapy approaches for the cure of beta-thalassemia and sickle cell anemia • treatments for disorders related to anemia of inflammation, hemochromatosis and polcythemia vera. We have many exciting ongoing and proposed projects, some of which are moving towards clinical trial. This position focuses on the gene therapy side of our lab, where you will learn to about our multiple genetic approaches to cure thalassemia and our preclinical lentiviral gene therapy vectors. Please see the work of Stefano Rivella and Laura Breda for more information.


Our laboratory is rapidly expanding, not only in the number of projects, but also in regards to the variety of international collaborations and the diversity that characterizes our personnel. We currently have researchers from the United States, Italy, China, Portugal, India, and Puerto Rico. We are part of an international consortium of laboratories studying thalassemia, and value a variety of individual experiences. We are looking for someone who is a good personal fit for our group, as this is absolutely crucial to us. We are a very social lab and enjoy a friendly, collegiate day-to-day atmosphere. Technical Skills preferred: -cell culture -previous work with mouse models, work with bone marrow transplants is a plus -basic molecular biology skills: RNA/DNA extraction, protein analyses, standard and quantitative PCR, genotyping, protein analysis via HPLC, also a big plus


Soft skills: -resourceful and extremely proactive -team player -ability to thrive in a collegial work environment -effective oral and written communication skills -outside interests Critical Thinking and Management skills: -hard working -self-motivated, flexible, able to take on a challenge -creative, problem solving -critical insight and genuine interest in the field and research

Full Address

University of Pennsylvania - Children's Hospital of Philadelphia
3615 Civic Center Boulevard
19104 Philadelphia, PA