ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Autor: Susan Tom, Kathleen Meyer, Kelly M. Podetz-Pedersen, Renee Cooksley, Michael C. Holmes, R. Scott McIvor, Russell Dekelver, Brenda Koniar, Michelle Rohde, Kanut Laoharawee, Scott Sproul, Chester B. Whitley, Susan St Martin, Robert Radeke, Li Ou, Yolanda Santiago, Thomas Wechsler, Michelle J. Przybilla

Publikováno v: Molecular Therapy

Mucopolysaccharidosis type I (MPS I) is a severe disease due to deficiency of the lysosomal hydrolase α-L-iduronidase (IDUA) and the subsequent accumulation of the glycosaminoglycans (GAG), leading to progressive, systemic disease and a shortened li

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::056906ea953e0f6ee7a887c3a7320cdb
http://europepmc.org/articles/PMC6319315

485. ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I

Autor: Susan Tom, R. Scott McIvor, Russell Dekelver, Li Ou, Chester B. Whitley, Michael J. Przybilla, Thomas Wechsler, Robert Radeke, Michael C. Holmes, Kanut Laoharawee, Renee Cooksley, Amy Manning-Bog, Brenda L. Komar, Scott Sproul, Michelle Rohde, Kelly M. Podetz-Pedersen

Publikováno v: Molecular Therapy. 24:S192-S193

Mucopolysaccharidosis type I (MPS I) is characterized by progressive neurodegeneration, and premature death (

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::2a33c6ef708aba0bdbed84d19b71f6dd
https://doi.org/10.1016/s1525-0016(16)33294-4

484. In Vivo Zinc-Finger Nuclease Mediated Iduronate-2-Sulfatase (IDS) Target Gene Insertion and Correction of Metabolic Disease in a Mouse Model of Mucopolysaccharidosis Type II (MPS II)

Autor: Kanut Laoharawee, Michelle Rohde, Amy Manning-Bog, Li Ou, Susan Tom, R. Scott McIvor, Kelly M. Podetz-Petersen, Kathleen Meyer, Russell Dekelver, Thomas Wechsler, Robert Radeke, Michael C. Holmes, Scott Sproul, Chester B. Whitley

Publikováno v: Molecular Therapy. 24:S192

Hunter syndrome (Mucopolysaccharidosis Type II, MPS II) is a rare X-linked lysosomal disorder caused by lack of functional iduronate-2 sulfatase (IDS) enzyme and subsequent accumulation of glycosaminoglycans (GAG) in affected individuals. Manifestati

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::3dff1a8d67035acac72f1cbb745ddfdd
https://doi.org/10.1016/s1525-0016(16)33293-2

ZFN-mediated correction of murine MPS I model by expression of the human IDUA cDNA from the albumin 'safe harbor' locus

Autor: Michelle Rhode, Kelly M. Podetz-Pedersen, Russell Dekelver, Michael J. Przybilla, Thomas Wechsler, Robert Radeke, Michael C. Holmes, Kanut Laoharawee, Susan Tom, Li Ou, Chester B. Whitley, Brenda Koniar, Renee Cooksley, Amy Manning-Bog, R. Scott McIvor, Scott Sproul

Publikováno v: Molecular Genetics and Metabolism. 117:S89

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::83fbcb4f486bf7470eaf60ce8192ab46
https://doi.org/10.1016/j.ymgme.2015.12.387

A new-generation stable inducible packaging cell line for lentiviral vectors

Autor: Luigi Naldini, Jin-Ping Song, Robert Radeke, Ryan Mcguinness, Deborah Farson, Rochelle Witt, Tom Dull, Anatoly Bukovsky, Michael C. Kelly, Antonella Consiglio

Publikováno v: HUMAN GENE THERAPY
r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
instname
HUM GENE THER
r-FSJD: Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
Fundació Sant Joan de Déu

We have successfully generated and characterized a stable packaging cell line for HIV-1-based vectors. To allow safe production of vector, a minimal packaging construct carrying only the coding sequences of the HIV-1 gag-pol, tat, and rev genes was s

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::05bd86042dbe9681a1c9d667af33f39a
https://pubmed.ncbi.nlm.nih.gov/11387062