| Year |
Citation |
Score |
| 2018 |
Donsante A, Boulis NM. Progress in gene and cell therapies for the neuronal ceroid lipofuscinoses. Expert Opinion On Biological Therapy. PMID 29936867 DOI: 10.1080/14712598.2018.1492544 |
0.421 |
|
| 2016 |
Wagner JL, O'Connor DM, Donsante A, Boulis NM. Gene, Stem Cell, and Alternative Therapies for SCA 1. Frontiers in Molecular Neuroscience. 9: 67. PMID 27570504 DOI: 10.3389/Fnmol.2016.00067 |
0.403 |
|
| 2016 |
Donsante A, McEachin Z, Riley J, Leung CH, Kanz L, O'Connor DM, Boulis NM. Intracerebroventricular delivery of self-complementary adeno-associated virus serotype 9 to the adult rat brain. Gene Therapy. PMID 26824881 DOI: 10.1038/Gt.2016.6 |
0.369 |
|
| 2016 |
McEachin ZT, Donsante A, Boulis N. Gene Therapy for the Treatment of Neurological Disorders: Amyotrophic Lateral Sclerosis. Methods in Molecular Biology (Clifton, N.J.). 1382: 399-408. PMID 26611602 DOI: 10.1007/978-1-4939-3271-9_28 |
0.429 |
|
| 2013 |
Haddad MR, Donsante A, Zerfas P, Kaler SG. Fetal Brain-directed AAV Gene Therapy Results in Rapid, Robust, and Persistent Transduction of Mouse Choroid Plexus Epithelia. Molecular Therapy. Nucleic Acids. 2: e101. PMID 23799375 DOI: 10.1038/Mtna.2013.27 |
0.443 |
|
| 2013 |
Donsante A, Sullivan P, Goldstein DS, Brinster LR, Kaler SG. L-threo-dihydroxyphenylserine corrects neurochemical abnormalities in a Menkes disease mouse model. Annals of Neurology. 73: 259-65. PMID 23224983 DOI: 10.1002/Ana.23787 |
0.353 |
|
| 2013 |
Schoonveld C, Donsante A, del Gaudio D, Waggoner D, Das S, Kaler SG. Prenatal diagnostic conundrum involving a novel ATP7A duplication. Clinical Genetics. 84: 97-8. PMID 23151012 DOI: 10.1111/Cge.12041 |
0.338 |
|
| 2012 |
Hicks JD, Donsante A, Pierson TM, Gillespie MJ, Chou DE, Kaler SG. Increased frequency of congenital heart defects in Menkes disease. Clinical Dysmorphology. 21: 59-63. PMID 22134099 DOI: 10.1097/Mcd.0B013E32834Ea52B |
0.335 |
|
| 2011 |
Donsante A, Yi L, Zerfas PM, Brinster LR, Sullivan P, Goldstein DS, Prohaska J, Centeno JA, Rushing E, Kaler SG. ATP7A gene addition to the choroid plexus results in long-term rescue of the lethal copper transport defect in a Menkes disease mouse model. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 2114-23. PMID 21878905 DOI: 10.1038/Mt.2011.143 |
0.458 |
|
| 2011 |
Desai V, Donsante A, Swoboda KJ, Martensen M, Thompson J, Kaler SG. Favorably skewed X-inactivation accounts for neurological sparing in female carriers of Menkes disease. Clinical Genetics. 79: 176-82. PMID 20497190 DOI: 10.1111/J.1399-0004.2010.01451.X |
0.309 |
|
| 2010 |
Donsante A, Johnson P, Jansen LA, Kaler SG. Somatic mosaicism in Menkes disease suggests choroid plexus-mediated copper transport to the developing brain. American Journal of Medical Genetics. Part A. 152: 2529-34. PMID 20799318 DOI: 10.1002/Ajmg.A.33632 |
0.343 |
|
| 2010 |
Kaler SG, Liew CJ, Donsante A, Hicks JD, Sato S, Greenfield JC. Molecular correlates of epilepsy in early diagnosed and treated Menkes disease. Journal of Inherited Metabolic Disease. 33: 583-9. PMID 20652413 DOI: 10.1007/S10545-010-9118-2 |
0.319 |
|
| 2009 |
Kaler SG, Tang J, Donsante A, Kaneski CR. Translational read-through of a nonsense mutation in ATP7A impacts treatment outcome in Menkes disease. Annals of Neurology. 65: 108-13. PMID 19194885 DOI: 10.1002/Ana.21576 |
0.346 |
|
| 2008 |
Tang J, Donsante A, Desai V, Patronas N, Kaler SG. Clinical outcomes in Menkes disease patients with a copper-responsive ATP7A mutation, G727R. Molecular Genetics and Metabolism. 95: 174-81. PMID 18752978 DOI: 10.1016/J.Ymgme.2008.06.015 |
0.358 |
|
| 2008 |
Kaler SG, Holmes CS, Goldstein DS, Tang J, Godwin SC, Donsante A, Liew CJ, Sato S, Patronas N. Neonatal diagnosis and treatment of Menkes disease. The New England Journal of Medicine. 358: 605-14. PMID 18256395 DOI: 10.1056/Nejmoa070613 |
0.354 |
|
| 2007 |
Donsante A, Miller DG, Li Y, Vogler C, Brunt EM, Russell DW, Sands MS. AAV vector integration sites in mouse hepatocellular carcinoma. Science (New York, N.Y.). 317: 477. PMID 17656716 DOI: 10.1126/Science.1142658 |
0.546 |
|
| 2007 |
Donsante A, Tang J, Godwin SC, Holmes CS, Goldstein DS, Bassuk A, Kaler SG. Differences in ATP7A gene expression underlie intrafamilial variability in Menkes disease/occipital horn syndrome. Journal of Medical Genetics. 44: 492-7. PMID 17496194 DOI: 10.1136/Jmg.2007.050013 |
0.335 |
|
| 2007 |
Donsante A, Levy B, Vogler C, Sands MS. Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII. Journal of Inherited Metabolic Disease. 30: 227-38. PMID 17308887 DOI: 10.1007/S10545-007-0483-4 |
0.563 |
|
| 2007 |
Lin D, Donsante A, Macauley S, Levy B, Vogler C, Sands MS. Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 44-52. PMID 17164774 DOI: 10.1038/Sj.Mt.6300026 |
0.592 |
|
| 2005 |
Lin D, Donsante A, Macauley S, Levy B, Vogler C, Sands M. 424. Intracranial AAV5 Synergizes with Non-Myeloablative Bone Marrow Transplantation in the Murine Model of Globoid-Cell Leukodystrophy Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.426 |
0.591 |
|
| 2005 |
Donsante A, Levy B, Vogler C, Sands M. 237. Evaluation of Low-Level, Constitutive Expression of |[beta]|-glucuronidase on the Clinical Manifestations of Mucopolysaccharidosis Type VII Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.240 |
0.596 |
|
| 2001 |
Donsante A, Vogler C, Muzyczka N, Crawford JM, Barker J, Flotte T, Campbell-Thompson M, Daly T, Sands MS. Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. Gene Therapy. 8: 1343-6. PMID 11571571 DOI: 10.1038/Sj.Gt.3301541 |
0.57 |
|
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