22 results on '"Rubin, Hillard"'
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2. Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH
3. AAVHSC characterization for developing treatments for human genetic diseases of the nervous system
4. Clade F AAVHSCs cross the blood brain barrier and transduce the central nervous system in addition to peripheral tissues following intravenous administration in nonhuman primates
5. Single intravenous dose of AAVHSC15 vector with human phenylalanine hydroxylase transgene results in sustained correction of phenylketonuria in the PAH mouse model
6. A Nonsense Mutation 1669Glu-->Ter within the Regulatory Domain of Human Erythroid Ankyrin Leads to a Selective Deficiency of the Major Ankyrin Isoform (Band 2.1) and a Phenotype of Autosomal Dominant Hereditary Spherocytosis
7. Duplication of 10 Nucleotides in the Erythroid Band 3 (AE1) Gene in a Kindred with Hereditary Spherocytosis and Band 3 Protein Deficiency (Band 3PRAGUE)
8. Low Seroprevalence of Neutralizing Antibodies Targeting Two Clade F AAV in Humans
9. Aurintricarboxylic acid increases yield of HSV-1 vectors
10. Inhibition of Choroidal Neovascularization in a Nonhuman Primate Model by Intravitreal Administration of an AAV2 Vector Expressing a Novel Anti-VEGF Molecule
11. 883. AAV-2 Based Gene Therapy Using Novel Anti-VEGF Molecules for Inhibition of Angiogenesis in the Eye
12. Bone Marrow Stromal Cell-Mediated Gene Therapy for Hemophilia A: In Vitro Expression of Human Factor VIII with High Biological Activity Requires the Inclusion of the Proteolytic Site at Amino Acid 1648
13. Autosomal Dominant Distal Renal Tubular Acidosis Is Associated in Three Families with Heterozygosity for the R589H Mutation in the AE1 (Band 3) Cl−/HCO3−Exchanger
14. The Structure and Organization of the Human Erythroid Anion Exchanger (AE1) Gene
15. Mutations of Conserved Arginines in the Membrane Domain of Erythroid Band 3 Lead to a Decrease in Membrane-Associated Band 3 and to the Phenotype of Hereditary Spherocytosis
16. Band 3 Tuscaloosa: Pro327→ Arg327Substitution in the Cytoplasmic Domain of Erythrocyte Band 3 Protein Associated With Spherocytic Hemolytic Anemia and Partial Deficiency of Protein 4.2
17. The Homozygous State for the Band 3 Protein Mutation in Southeast Asian Ovalocytosis May Be Lethal
18. Characterization of 13 Novel Band 3 Gene Defects in Hereditary Spherocytosis With Band 3 Deficiency
19. Band 3 Tuscaloosa: Pro327 → Arg327 Substitution in the Cytoplasmic Domain of Erythrocyte Band 3 Protein Associated With Spherocytic Hemolytic Anemia and Partial Deficiency of Protein 4.2
20. Band 3 Memphis: A Widespread Polymorphism With Abnormal Electrophoretic Mobility of Erythrocyte Band 3 Protein Caused by Substitution AAG → GAG (Lys → Glu) in Codon 56
21. Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH.
22. Aurintricarboxylic acid increases yield of HSV-1 vectors.
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