High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease

Alglucosidase alfa: Long term use in the treatment of patients with Pompe disease

Michael Beck

September 2009

Michael BeckChildren&rsquo;s Hospital, University of Mainz, Mainz, GermanyAbstract: Pompe diseas...

Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk.

Hout, J.M. van den
Reuser, A.J.J.
Klerk, J.B.C. de
Arts, W.F.M.
Smeitink, J.A.M.
Ploeg, A.T. van der

January 2001

Pompe disease is a metabolic myopathy caused by deficiency of lysosomal acid alpha-glucosidase. In t...

Recombinant human acid alpha-glucosidase corrects acid alpha-glucosidase-deficient human fibroblasts, quail fibroblasts, and quail myoblasts

Yang, H. W.

[[sponsorship]]生物醫學科學研究所[[note]]已出版;[SCI];沒有審查制度;具代表性[[note]]http://gateway.isiknowledge.com/gateway...

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease

Kishnani, P. S.

[[sponsorship]]生物醫學科學研究所[[note]]已出版;沒有審查制度;具代表性[[note]]http://gateway.isiknowledge.com/gateway/Gatew...

Replacing acid is proportional to-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers

Raben, N.
Fukuda, T.
Gilbert, A.
de Jong, D.
Thurberg, B.
Mattaliano, R.
Meikle, P.
Hopwood, J.
Nagashima, K.
Nagaraju, K.
Plotz, P.

January 2005

Pompe disease (type II glycogen storage disease) is an autosomal recessive disorder caused by a defi...

Long-term enzyme replacement therapy for Pompe disease with recombinant human alpha-glucosidase derived from chinese hamster ovary cells.

ROSSI M
ROSAPEPE F
PARENTI, GIANCARLO
DELLA CASA, ROBERTO
ROMANO, ALFONSO
MANSI, GIUSEPPINA
AGOVINO, teresa
VOSA, CARLO
DEL GIUDICE, ENNIO
ANDRIA, GENEROSO

January 2007

Pompe disease is a rare autosomal recessive myopathy due to the deficiency of lysosomal acid alpha-g...

Isolation and characterisation of a recombinant precursor form of lysosomal acid alpha-glucosidase

Fuller, M.
Van der Ploeg, A.
Reuser, A.
Anson, D.
Hopwood, J.

January 1995

Glycogenosis type II (GSD II, Pompe disease) is an autosomal recessive lysosomal storage disease tha...

Recombinant human acid alpha-glucosidase. Major clinical benefits in infantile-onset Pompe disease

Kishnani, P. S.

[[sponsorship]]生物醫學科學研究所[[note]]已出版;[SCI];有審查制度;具代表性[[note]]http://gateway.isiknowledge.com/gateway/...

The Pharmacological Chaperone AT2220 Increases the Specific Activity and Lysosomal Delivery of Mutant Acid Alpha-Glucosidase, and Promotes Glycogen Reduction in a Transgenic Mouse Model of Pompe Disease

Richie Khanna (150496)
Allan C. Powe Jr (600820)
Yi Lun (150519)
Rebecca Soska (150506)
Jessie Feng (150503)
Rohini Dhulipala (600821)
Michelle Frascella (150509)
Anadina Garcia (600822)
Lee J. Pellegrino (150514)
Su Xu (1317807)
Nastry Brignol (398532)
Matthew J. Toth (600823)
Hung V. Do (600824)
David J. Lockhart (150528)
Brandon A. Wustman (600825)
Kenneth J. Valenzano (150531)

July 2014

<div><p>Pompe disease is an inherited lysosomal storage disorder that results from a deficiency in a...

Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype

Til, Niek
Stok, Merel
Aerts, Fatima
Waard, Monique
Farahbakhshian, Elnaz
Visser, Trui
Haan, Marian
Jacobs, Ed
Willart, MAM
van der Wegen, CG (Pascal)
Scholte, Bob
Lambrecht, Bart
Duncker, Dirk-jan
van der Ploeg, Ans
Reuser, Arnold
Verstegen, Monique
Wagemaker, Gerard

January 2010

Pompe disease (acid alpha-glucosidase deficiency) is a lysosomal glycogen storage disorder character...

Enzyme Therapy in Non-classic Pompe’s Disease: Safety and efficacy of recombinant human a-glucosidase from milk of transgenic rabbits and from Chinese hamster ovary cells

Winkel, Léon

November 2004

textabstractPompe’s disease is an inherited metabolic illness, caused by an inherited deficiency of...

Generalized glycogen storage and cardiomegaly in a knockout mouse model of Pompe disease

Bijvoet, Agnes
Ploeg, Ans
Kamp, E.H.
Kroos, Marian
Ding, Jia-Huan
Yang, Bing
Visser, Pim
Bakker, Cathy
Verbeet, Martin
Oostra, Ben
Reuser, Arnold

January 1998

textabstractGlycogen storage disease type II (GSDII; Pompe disease), caused by inherited ...

Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapy

Raben, N.
Lu, N.
Nagaraju, K.
Rivera, Y.
Lee, A.
Yan, B.
Byrne, B.
Meikle, P.
Umapathysivam, K.
Hopwood, J.
Plotz, P.

January 2001

Both enzyme replacement and gene therapy of lysosomal storage disorders rely on the receptor-mediate...

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid alpha-glucosidase

Meliani, A.
Cohen-Tannoudji, M.
Astord, S.
Gjata, B.
Sellier, P.
Wittenberghe, L., Van
Vignaud, A.
Boisgerault, F.
Barkats, M.
Laforet, P.
Kay, M. A.
Koeberl, D. D.
Ronzitti, G.
Puzzo, F.
Colella, P.
Biferi, M. G.
Bali, D.
Paulk, N. K.
Vidal, P.
Collaud, F.
Simon-Sola, M.
Charles, S.
Hardet, R.
Leborgne, C.
Mingozzi, F.

November 2017

International audienceGlycogen storage disease type II or Pompe disease is a severe neuromuscular di...

Restoration of muscle functionality by genetic suppression of glycogen synthesis in a murine model of Pompe disease

Douillard-Guilloux, Gaelle
Raben, Nina
Takikita, Shoichi
Ferry, Arnaud
Vignaud, Alban
Guillet-Deniau, Isabelle
Favier, Maryline
Thurberg, Beth L.
Roach, Peter J.
Caillaud, Catherine
Richard, Emmanuel

February 2010

Glycogen storage disease type II (GSDII) or Pompe disease is an autosomal recessive disorder caused ...

Alglucosidase alfa: Long term use in the treatment of patients with Pompe disease

Michael Beck

September 2009

Michael BeckChildren&rsquo;s Hospital, University of Mainz, Mainz, GermanyAbstract: Pompe diseas...

Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk.

Hout, J.M. van den
Reuser, A.J.J.
Klerk, J.B.C. de
Arts, W.F.M.
Smeitink, J.A.M.
Ploeg, A.T. van der

January 2001

Pompe disease is a metabolic myopathy caused by deficiency of lysosomal acid alpha-glucosidase. In t...

Recombinant human acid alpha-glucosidase corrects acid alpha-glucosidase-deficient human fibroblasts, quail fibroblasts, and quail myoblasts

Yang, H. W.

[[sponsorship]]生物醫學科學研究所[[note]]已出版;[SCI];沒有審查制度;具代表性[[note]]http://gateway.isiknowledge.com/gateway...

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease

Kishnani, P. S.

[[sponsorship]]生物醫學科學研究所[[note]]已出版;沒有審查制度;具代表性[[note]]http://gateway.isiknowledge.com/gateway/Gatew...

Replacing acid is proportional to-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers

Raben, N.
Fukuda, T.
Gilbert, A.
de Jong, D.
Thurberg, B.
Mattaliano, R.
Meikle, P.
Hopwood, J.
Nagashima, K.
Nagaraju, K.
Plotz, P.

January 2005

Pompe disease (type II glycogen storage disease) is an autosomal recessive disorder caused by a defi...

Long-term enzyme replacement therapy for Pompe disease with recombinant human alpha-glucosidase derived from chinese hamster ovary cells.

ROSSI M
ROSAPEPE F
PARENTI, GIANCARLO
DELLA CASA, ROBERTO
ROMANO, ALFONSO
MANSI, GIUSEPPINA
AGOVINO, teresa
VOSA, CARLO
DEL GIUDICE, ENNIO
ANDRIA, GENEROSO

January 2007

Pompe disease is a rare autosomal recessive myopathy due to the deficiency of lysosomal acid alpha-g...

Isolation and characterisation of a recombinant precursor form of lysosomal acid alpha-glucosidase

Fuller, M.
Van der Ploeg, A.
Reuser, A.
Anson, D.
Hopwood, J.

January 1995

Glycogenosis type II (GSD II, Pompe disease) is an autosomal recessive lysosomal storage disease tha...

Recombinant human acid alpha-glucosidase. Major clinical benefits in infantile-onset Pompe disease

Kishnani, P. S.

[[sponsorship]]生物醫學科學研究所[[note]]已出版;[SCI];有審查制度;具代表性[[note]]http://gateway.isiknowledge.com/gateway/...

The Pharmacological Chaperone AT2220 Increases the Specific Activity and Lysosomal Delivery of Mutant Acid Alpha-Glucosidase, and Promotes Glycogen Reduction in a Transgenic Mouse Model of Pompe Disease

Richie Khanna (150496)
Allan C. Powe Jr (600820)
Yi Lun (150519)
Rebecca Soska (150506)
Jessie Feng (150503)
Rohini Dhulipala (600821)
Michelle Frascella (150509)
Anadina Garcia (600822)
Lee J. Pellegrino (150514)
Su Xu (1317807)
Nastry Brignol (398532)
Matthew J. Toth (600823)
Hung V. Do (600824)
David J. Lockhart (150528)
Brandon A. Wustman (600825)
Kenneth J. Valenzano (150531)

July 2014

<div><p>Pompe disease is an inherited lysosomal storage disorder that results from a deficiency in a...

Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype

Til, Niek
Stok, Merel
Aerts, Fatima
Waard, Monique
Farahbakhshian, Elnaz
Visser, Trui
Haan, Marian
Jacobs, Ed
Willart, MAM
van der Wegen, CG (Pascal)
Scholte, Bob
Lambrecht, Bart
Duncker, Dirk-jan
van der Ploeg, Ans
Reuser, Arnold
Verstegen, Monique
Wagemaker, Gerard

January 2010

Pompe disease (acid alpha-glucosidase deficiency) is a lysosomal glycogen storage disorder character...

Enzyme Therapy in Non-classic Pompe’s Disease: Safety and efficacy of recombinant human a-glucosidase from milk of transgenic rabbits and from Chinese hamster ovary cells

Winkel, Léon

November 2004

textabstractPompe’s disease is an inherited metabolic illness, caused by an inherited deficiency of...

Generalized glycogen storage and cardiomegaly in a knockout mouse model of Pompe disease

Bijvoet, Agnes
Ploeg, Ans
Kamp, E.H.
Kroos, Marian
Ding, Jia-Huan
Yang, Bing
Visser, Pim
Bakker, Cathy
Verbeet, Martin
Oostra, Ben
Reuser, Arnold

January 1998

textabstractGlycogen storage disease type II (GSDII; Pompe disease), caused by inherited ...

Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapy

Raben, N.
Lu, N.
Nagaraju, K.
Rivera, Y.
Lee, A.
Yan, B.
Byrne, B.
Meikle, P.
Umapathysivam, K.
Hopwood, J.
Plotz, P.

January 2001

Both enzyme replacement and gene therapy of lysosomal storage disorders rely on the receptor-mediate...

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid alpha-glucosidase

Meliani, A.
Cohen-Tannoudji, M.
Astord, S.
Gjata, B.
Sellier, P.
Wittenberghe, L., Van
Vignaud, A.
Boisgerault, F.
Barkats, M.
Laforet, P.
Kay, M. A.
Koeberl, D. D.
Ronzitti, G.
Puzzo, F.
Colella, P.
Biferi, M. G.
Bali, D.
Paulk, N. K.
Vidal, P.
Collaud, F.
Simon-Sola, M.
Charles, S.
Hardet, R.
Leborgne, C.
Mingozzi, F.

November 2017

International audienceGlycogen storage disease type II or Pompe disease is a severe neuromuscular di...

Restoration of muscle functionality by genetic suppression of glycogen synthesis in a murine model of Pompe disease

Douillard-Guilloux, Gaelle
Raben, Nina
Takikita, Shoichi
Ferry, Arnaud
Vignaud, Alban
Guillet-Deniau, Isabelle
Favier, Maryline
Thurberg, Beth L.
Roach, Peter J.
Caillaud, Catherine
Richard, Emmanuel

February 2010

Glycogen storage disease type II (GSDII) or Pompe disease is an autosomal recessive disorder caused ...

Alglucosidase alfa: Long term use in the treatment of patients with Pompe disease

Michael Beck

September 2009

Michael BeckChildren&rsquo;s Hospital, University of Mainz, Mainz, GermanyAbstract: Pompe diseas...

Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk.

Hout, J.M. van den
Reuser, A.J.J.
Klerk, J.B.C. de
Arts, W.F.M.
Smeitink, J.A.M.
Ploeg, A.T. van der

January 2001

Pompe disease is a metabolic myopathy caused by deficiency of lysosomal acid alpha-glucosidase. In t...

Recombinant human acid alpha-glucosidase corrects acid alpha-glucosidase-deficient human fibroblasts, quail fibroblasts, and quail myoblasts

Yang, H. W.

[[sponsorship]]生物醫學科學研究所[[note]]已出版;[SCI];沒有審查制度;具代表性[[note]]http://gateway.isiknowledge.com/gateway...

High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease

Abstract

Extracted data

High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease

Abstract

Extracted data

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