Neeltje A Kootstra
Affiliation: The Salk Institute
- Gene therapy with viral vectorsNeeltje A Kootstra
Laboratory of Genetics, The Salk Institute, La Jolla, California 92037 1099, USA
Annu Rev Pharmacol Toxicol 43:413-39. 2003..Even though the results of these trials are encouraging, vector development is still required to improve and refine future treatment of hereditary disorders...
- Efficient production of human FVIII in hemophilic mice using lentiviral vectorsNeeltje A Kootstra
Laboratory of Genetics, The Salk Institute, La Jolla, California 92037, USA
Mol Ther 7:623-31. 2003..The challenge is to overcome the immune barriers to the therapeutic gene product...
- Abrogation of postentry restriction of HIV-1-based lentiviral vector transduction in simian cellsNeeltje A Kootstra
Laboratory of Genetics and Infectious Disease Laboratory, The Salk Institute, La Jolla, CA 92037 1099, USA
Proc Natl Acad Sci U S A 100:1298-303. 2003..Overall, the findings demonstrate that the host range of HIV-1-based LV can be altered by modifications in the packaging construct...
- Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypesTippi C MacKenzie
Children s Institute for Surgical Science, Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
Mol Ther 6:349-58. 2002..m. and i.h. injection of all three vectors. Our findings of long-term transduction of skeletal myocytes and cardiomyocytes after in utero administration suggest a novel strategy for the treatment of congenital muscular dystrophies...
- Efficient transduction of simian cells by HIV-1-based lentiviral vectors that contain mutations in the capsid proteinMaarten A N Rits
Department of Clinical Viro Immunology, Sanquin Research, Landsteiner Laboratory, Amsterdam, The Netherlands
Mol Ther 15:930-7. 2007..Therefore, this LV might be very useful for long-term safety studies in large animal models like rhesus macaques...