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Expert Opinion on Investigational Drugs|July 5, 2005
Gene therapy for lysosomal storage diseasesT M Daly, M S SandsPediatric Research|June 1, 2000
Abnormal immune function in vivo in a murine model of lysosomal storage diseaseT M Daly, R G Lorenz, M S SandsProceedings of the National Academy of Sciences of the United States of America|March 3, 1999
Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage diseaseT M Daly, C Vogler, B Levy, et al.Human Gene Therapy|February 18, 1999
Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII miceT M Daly, T Okuyama, C Vogler, et al.Gene Therapy|September 26, 2001
Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transferT M Daly, K K Ohlemiller, M S Roberts, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|March 29, 2001
Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VIIW A Frisella, L H O'Connor, C A Vogler, et al.Nucleic Acids Research|June 11, 1992
Binding of TFIIIA to derivatives of 5S RNA containing sequence substitutions or deletions defines a minimal TFIIIA binding siteD F Bogenhagen, M S SandsMolecular and Cellular Biology|November 1, 1987
TFIIIA binds to different domains of 5S RNA and the Xenopus borealis 5S RNA geneM S Sands, D F BogenhagenProceedings of the National Academy of Sciences of the United States of America|July 15, 1993
A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VIIM S Sands, E H BirkenmeierNucleic Acids Research|April 25, 1991
The carboxyterminal zinc fingers of TFIIIA interact with the tip of helix V of 5S RNA in the 7S ribonucleoprotein particleM S Sands, D F BogenhagenPageof 7