H H Stedman
Affiliation: University of Pennsylvania
- Myosin gene mutation correlates with anatomical changes in the human lineageHansell H Stedman
Department of Surgery, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
Nature 428:415-8. 2004..This represents the first proteomic distinction between humans and chimpanzees that can be correlated with a traceable anatomic imprint in the fossil record...
- Molecular approaches to therapy for Duchenne and limb-girdle muscular dystrophyH H Stedman
Department of Surgery and Institute for Human Gene Therapy, University of Pennsylvania School of Medicine, Philadelphia 19104 6160, USA
Curr Opin Mol Ther 3:350-6. 2001....
- Sarcomeres are added in series to emphysematous rat diaphragm after lung volume reduction surgeryJoseph B Shrager
Department of Surgery, The University of Pennsylvania School of Medicine, Philadelphia, PA, USA
Chest 121:210-5. 2002..We used a model of elastase-induced emphysema in rats to test the hypothesis that sarcomere addition occurs following LVRS...
- Evolutionary implications of three novel members of the human sarcomeric myosin heavy chain gene familyPhilippe R Desjardins
Department of Surgery, School of Medicine, University of Pennsylvania, 421 Curie Blvd, Philadelphia, PA 19104, USA
Mol Biol Evol 19:375-93. 2002..One of the novel human genes is found to have introns at positions shared only with the molluscan catchin/MyHC gene, providing evidence for the structure of a pre-Cambrian ancestral gene...
- Myosin heavy chain and physiological adaptation of the rat diaphragm in elastase-induced emphysemaDong Kwan Kim
Department of Surgery, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA
Respir Res 4:1. 2003....
- A PCR-based assay for the wild-type dystrophin gene transferred into the mdx mouseJ B Shrager
Department of Surgery, University of Pennsylvania School of Medicine, Philadelphia 19104
Muscle Nerve 15:1133-7. 1992..This simple technique should allow a number of basic questions related to myoblast and direct gene transfer to be addressed using the mdx mouse model...
- Lung volume reduction surgery restores the normal diaphragmatic length-tension relationship in emphysematous ratsJ B Shrager
Section of General Thoracic Surgery, Department of Surgery, Philadelphia Veterans Affairs Medical Center, Philadelphia, PA 19104, USA
J Thorac Cardiovasc Surg 121:217-24. 2001..We studied length adaptation in rat diaphragmatic muscle in an attempt to elucidate the mechanism by which diaphragmatic function improves after this controversial operation...
- Myocardial gene delivery using molecular cardiac surgery with recombinant adeno-associated virus vectors in vivoJ D White
Division of Cardiovascular Surgery, Department of Surgery, University of Pennsylvania School of Medicine, Philadelphia, PA 19104, USA
Gene Ther 18:546-52. 2011....
- Inspiratory loading does not accelerate dystrophy in mdx mouse diaphragm: implications for regenerative therapyAlexander S Krupnick
Department of Surgery, University of Pennsylvania School of Medicine, Philadelphia 19104, USA
J Appl Physiol 94:411-9. 2003....
- Global cardiac-specific transgene expression using cardiopulmonary bypass with cardiac isolationCharles R Bridges
Department of Surgery, University of Pennsylvania Medical Center, Philadelphia, USA
Ann Thorac Surg 73:1939-46. 2002..To address this problem, we developed a novel cannulation technique that achieves efficient isolation of the heart in situ using separate cardiopulmonary bypass (CPB) circuits for the heart and body in dogs...
- Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal modelValder R Arruda
The Children s Hospital of Philadelphia, 3615 Civic Center Blvd, 302 Abramson Research Center, Philadelphia, PA 19104, USA
Blood 105:3458-64. 2005..These findings also have immediate relevance for gene transfer in patients with muscular dystrophy...
- Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vectorLeonard T Su
Department of Surgery, University of Pennsylvania, Philadelphia, PA 19104, USA
Circulation 112:1780-8. 2005..We provide the first demonstration of uniform gene transfer to muscle fibers of an entire extremity in the dog, providing a firm foundation for further translational studies of efficacy in canine models for human diseases...
- Efficient myocyte gene delivery with complete cardiac surgical isolation in situCharles R Bridges
Department of Surgery of the University of Pennsylvania Health System, Philadelphia, PA 19104, USA
J Thorac Cardiovasc Surg 130:1364. 2005....
- Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia BValder R Arruda
University of Pennsylvania School of Medicine, Philadelphia, PA, USA
Blood 115:4678-88. 2010..Collectively, these results demonstrate the feasibility of this approach for treatment of HB and highlight the importance of IS to prevent immune responses to the FIX transgene product...
- Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogsVirginia Haurigot
Division of Hematology and Center for Cellular and Molecular Therapeutics, Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
Mol Ther 18:1318-29. 2010..In summary, this study in a large animal model of HB demonstrates that therapeutic levels of gene transfer can be safely achieved using a novel route of intravascular gene transfer to muscle...
- Surgical Approaches to Systemic Gene TransferHansell Stedman; Fiscal Year: 2006..It will also provide specific information about the rational design of strategies for systemic gene therapy in one of the most common single-gene lethal diseases in man, Duchenne Muscular Dystrophy. ..
- Translational program for molecular therapeutics in DMDHansell Stedman; Fiscal Year: 2007....
- Systemic molecular therapy for muscular dystrophyHansell H Stedman; Fiscal Year: 2010..We use a novel approach to direct gene transfer to replace the missing dystrophin protein in locomotive, respiratory and cardiac muscle in the dystrophic dog, and use a prospective, randomized study to evaluate therapeutic efficacy. ..