Correction/mutation of acid α-D-glucosidase gene by modified single-stranded oligonucleotides: in vitro and in vivo studies
暂无分享,去创建一个
[1] J. Smeitink,et al. Enzyme therapy for Pompe disease with recombinant human α-glucosidase from rabbit milk , 2001, Journal of Inherited Metabolic Disease.
[2] M. Tan,et al. Expression of a Functional Asialoglycoprotein Receptor in Human Renal Proximal Tubular Epithelial Cells , 2002, Nephron.
[3] M. Eller,et al. DNA oligonucleotide treatment corrects the age‐associated decline in DNA repair capacity , 2002, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.
[4] M. Rice,et al. In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides. , 2001, Nucleic acids research.
[5] E. Kmiec,et al. Targeted Gene Repair in Mammalian Cells Using Chimeric RNA/DNA Oligonucleotides and Modified Single-Stranded Vectors , 2001, Science's STKE.
[6] O. Igoucheva,et al. Targeted gene correction by small single-stranded oligonucleotides in mammalian cells , 2001, Gene Therapy.
[7] J. Smeitink,et al. Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk. , 2001, Journal of inherited metabolic disease.
[8] S. Tsujino,et al. [Acid maltase deficiency]. , 2001, Ryoikibetsu shokogun shirizu.
[9] J. Charrow,et al. Recombinant human acid α-glucosidase enzyme therapy for infantile glycogen storage disease type II: Results of a phase I/II clinical trial , 2001, Genetics in Medicine.
[10] A. Vulto,et al. Recombinant human α-glucosidase from rabbit milk in Pompe patients , 2000, The Lancet.
[11] Shui-Tein Chen,et al. Facile Solid Phase Synthesis of YEE(ah-GalNAc)3, a Ligand with Known High Affinity for the Asialoglycoprotein Receptor , 2000 .
[12] A. Reuser,et al. Recombinant human alpha-glucosidase from rabbit milk in Pompe patients. , 2000, Lancet.
[13] N. Raben,et al. Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase. , 1999, Proceedings of the National Academy of Sciences of the United States of America.
[14] J. Metzl,et al. An Interesting Case of Infant Sudden Death: Severe Hypertrophic Cardiomyopathy in Pompe's Disease , 1999, Pacing and clinical electrophysiology : PACE.
[15] A. Amalfitano,et al. Persistence of an [E1-, polymerase-] adenovirus vector despite transduction of a neoantigen into immune-competent mice. , 1999, Human gene therapy.
[16] A. Kahn,et al. Adenovirus-mediated transfer of the acid alpha-glucosidase gene into fibroblasts, myoblasts and myotubes from patients with glycogen storage disease type II leads to high level expression of enzyme and corrects glycogen accumulation. , 1998, Human molecular genetics.
[17] J. V. Van Hove,et al. Recombinant human acid alpha-glucosidase corrects acid alpha-glucosidase-deficient human fibroblasts, quail fibroblasts, and quail myoblasts. , 1998, Pediatric research.
[18] J. Shieh,et al. Molecular study on the infantile form of Pompe disease in Chinese in Taiwan. , 1996, Zhonghua Minguo xiao er ke yi xue hui za zhi [Journal]. Zhonghua Minguo xiao er ke yi xue hui.
[19] J. Levis,et al. Cell-type specific and ligand specific enhancement of cellular uptake of oligodeoxynucleoside methylphosphonates covalently linked with a neoglycopeptide, YEE(ah-GalNAc)3. , 1995, Bioconjugate chemistry.
[20] K. Zatloukal,et al. Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand. , 1992, Bioconjugate chemistry.
[21] F. Sherman,et al. Strand-specificity in the transformation of yeast with synthetic oligonucleotides. , 1992, Genetics.
[22] M. Spiess,et al. Asialoglycoprotein receptor. , 1992, International review of cytology.
[23] F. Sherman,et al. Transformation of yeast with synthetic oligonucleotides. , 1988, Proceedings of the National Academy of Sciences of the United States of America.
[24] K. Hsiao,et al. Study of alpha-D-glucosidase activity in patients with Pompe's disease. , 1986, Taiwan yi xue hui za zhi. Journal of the Formosan Medical Association.
[25] J. Martín,et al. Identification of heterozygotes for glycogenosis 2 (Acid maltase deficiency) , 1981, Clinical genetics.
[26] P. Gillette,et al. Electrophysiological mechanism of the short PR interval in Pompe disease. , 1974, American journal of diseases of children.
[27] H. Nadler,et al. Alpha-1,4 glucosidase activity in Pompe's disease. , 1971, The Journal of pediatrics.
[28] H. Hers. alpha-Glucosidase deficiency in generalized glycogenstorage disease (Pompe's disease). , 1963, The Biochemical journal.
[29] H. Hers. α-Glucosidase deficiency in generalized glycogen-storage disease (Pompe's disease) , 1963 .