Search results Cooksley, Renee D.

1.	ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome Ou, Li; DeKelver, Russell C.; Rohde, Michelle ... Molecular therapy, 01/2019, Volume: 27, Issue: 1 Journal Article Peer reviewed Open access 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 ...	Full text Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UILJ, UL, UM, UPCLJ, UPUK, ZAGLJ, ZRSKP PDF
2.	Enzyme replacement therapy with α-L-iduronidase and lectin RTB fusion protein in treating murine Hurler syndrome Ou, Li; Acosta, Walter; Koniar, Brenda L. ... Molecular genetics and metabolism, February 2016, 2016-02-00, Volume: 117, Issue: 2 Journal Article Peer reviewed Open access	Full text Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK
3.	ZFN-mediated in vivo genome editing of hepatocytes results in phenotypic correction in murine MPS I and MPS II models Wechsler, Thomas; DeKelver, Russell; Laoharawee, Kanut ... Molecular genetics and metabolism, February 2018, 2018-02-00, Volume: 123, Issue: 2 Journal Article Peer reviewed	Full text Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK
4.	485. ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I Ou, Li; DeKelver, Russell; Tom, Susan ... Molecular therapy, 20/May , Volume: 24 Journal Article Peer reviewed Open access	Full text Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UILJ, UL, UM, UPCLJ, UPUK, ZAGLJ, ZRSKP PDF
5.	ZFN-mediated in vivo genome editing results in phenotypic correction in murine MPS I and MPS II models DeKelver, Russell; Ou, Li; Laoharawee, Kanut ... Molecular genetics and metabolism, January-February 2017, 2017-01-00, Volume: 120, Issue: 1-2 Journal Article Peer reviewed	Full text Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK
6.	ZFN-mediated correction of murine MPS I model by expression of the human IDUA cDNA from the albumin “safe harbor” locus Ou, Li; DeKelver, Russell; Tom, Susan ... Molecular genetics and metabolism, February 2016, 2016-02-00, Volume: 117, Issue: 2 Journal Article Peer reviewed	Full text Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK, ZRSKP
7.	Buccal Swab Specimens for IDUA Genotype Analysis for Patients after Hematopoietic Stem Cell Transplantation Cooksley, Renee D.; Shapiro, Elsa; Orchard, Paul ... Molecular genetics and metabolism, 02/2012, Volume: 105, Issue: 2 Journal Article Peer reviewed	Full text Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK
8.	28. Case studies in mutational analysis: Challenges remain Cooksley, Renee; Sandberg, Sherri; Score, Paul ... Molecular genetics and metabolism, 02/2009, Volume: 96, Issue: 2 Journal Article Peer reviewed	Full text Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK

1.

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome
Ou, Li; DeKelver, Russell C.; Rohde, Michelle ... Molecular therapy, 01/2019, Volume: 27, Issue: 1
Journal Article

Peer reviewed

Open access

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 ...

Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UILJ, UL, UM, UPCLJ, UPUK, ZAGLJ, ZRSKP

PDF

2.

Enzyme replacement therapy with α-L-iduronidase and lectin RTB fusion protein in treating murine Hurler syndrome
Ou, Li; Acosta, Walter; Koniar, Brenda L. ... Molecular genetics and metabolism, February 2016, 2016-02-00, Volume: 117, Issue: 2
Journal Article

Peer reviewed

Open access

Full text

Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK

3.

ZFN-mediated in vivo genome editing of hepatocytes results in phenotypic correction in murine MPS I and MPS II models
Wechsler, Thomas; DeKelver, Russell; Laoharawee, Kanut ... Molecular genetics and metabolism, February 2018, 2018-02-00, Volume: 123, Issue: 2
Journal Article

Peer reviewed

Full text

Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK

4.

485. ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I
Ou, Li; DeKelver, Russell; Tom, Susan ... Molecular therapy, 20/May , Volume: 24
Journal Article

Peer reviewed

Open access

Full text

Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UILJ, UL, UM, UPCLJ, UPUK, ZAGLJ, ZRSKP

PDF

5.

ZFN-mediated in vivo genome editing results in phenotypic correction in murine MPS I and MPS II models
DeKelver, Russell; Ou, Li; Laoharawee, Kanut ... Molecular genetics and metabolism, January-February 2017, 2017-01-00, Volume: 120, Issue: 1-2
Journal Article

Peer reviewed

Full text

Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK

6.

ZFN-mediated correction of murine MPS I model by expression of the human IDUA cDNA from the albumin “safe harbor” locus
Ou, Li; DeKelver, Russell; Tom, Susan ... Molecular genetics and metabolism, February 2016, 2016-02-00, Volume: 117, Issue: 2
Journal Article

Peer reviewed

Full text

Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK, ZRSKP

7.

Buccal Swab Specimens for IDUA Genotype Analysis for Patients after Hematopoietic Stem Cell Transplantation
Cooksley, Renee D.; Shapiro, Elsa; Orchard, Paul ... Molecular genetics and metabolism, 02/2012, Volume: 105, Issue: 2
Journal Article

Peer reviewed

Full text

Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK

8.

28. Case studies in mutational analysis: Challenges remain
Cooksley, Renee; Sandberg, Sherri; Score, Paul ... Molecular genetics and metabolism, 02/2009, Volume: 96, Issue: 2
Journal Article

Peer reviewed

Full text

Available for: GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK

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