α glucosidase recombinant human gaa myozyme  (Genzyme)

Journal: Clinical and Translational Medicine

Article Title: Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease

doi: 10.1002/ctm2.70314

Figure Lengend Snippet: Model of pharmacological intervention in Pompe disease combining GYS1 ASO‐based SRT and ERT. Proposed adjunct therapeutic approach for Pompe disease based on the reduction of the synthesis of new glycogen via GYS1 ASO‐mediated SRT, combined with the ERT‐mediated clearance of preexisting glycogen that has accumulated in the lysosome because of GAA inactivation. Such combinatorial strategy could potentially result in greater benefit for Pompe patients, especially in skeletal muscle, where ERT alone is not remarkably effective. SRT: substrate reduction therapy; ERT: enzyme replacement therapy; GYS1: glycogen synthase 1; ASO: antisense oligonucleotide; GAA: glucosidase, alpha acid; rhGAA: recombinant human GAA.

Article Snippet: Starting from week 7 the mice additionally received 20 mg/kg recombinant human GAA (rhGAA) (Genzyme Corporation, Cambridge, MA; Cat.: NDC 58468‐0160‐1) via intravenous injection once every 2 weeks.

Techniques: Recombinant

Journal: bioRxiv

Article Title: Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease

doi: 10.1101/2024.02.22.580414

Figure Lengend Snippet: We propose an adjunct therapeutic approach for Pompe disease based on the reduction of the synthesis of new glycogen via GYS1 ASO-mediated SRT, combined with the ERT-mediated clearance of pre-existing glycogen that has accumulated in the lysosome because of GAA inactivation. Such combinatorial strategy would result in greater clinical benefit for Pompe patients, especially in skeletal muscle, where ERT alone is not remarkably effective. SRT: substrate reduction therapy; ERT: enzyme replacement therapy; GYS1: glycogen synthase 1; ASO: antisense oligonucleotide; GAA: glucosidase, alpha acid; rhGAA: recombinant human GAA.

Article Snippet: Starting from week 7 the mice additionally received 20 mg/kg recombinant human GAA (rhGAA) (Genzyme Corporation, Cambridge, MA; Cat.: NDC 58468-0160-1) via intravenous injection once every two weeks.

Techniques: Recombinant

Journal: bioRxiv

Article Title: Preclinical studies with ground germinated barley (GGB) for oral enzyme replacement therapy (Oral-ERT) in Pompe disease knockout mice

doi: 10.1101/2023.12.27.573457

Figure Lengend Snippet: is a bar graph showing human PD lymphoid lines exposed to mock treatment, bGAA 50-100kD fraction, human placental GAA or rhGAA for 48 hours and assayed for GAA. All treatments were significant at p = <0.05.

Article Snippet: ERT with a recombinant human GAA (rhGAA) secreted by CHO cells (alglucosidase alfa/Myozyme/Lumizyme, Genzyme/Sanofi Corp.) infused every two weeks is the first approved therapy.

Techniques:

Journal: bioRxiv

Article Title: Preclinical studies with ground germinated barley (GGB) for oral enzyme replacement therapy (Oral-ERT) in Pompe disease knockout mice

doi: 10.1101/2023.12.27.573457

Figure Lengend Snippet: is a bar graph showing human fibroblast cell lines human fibroblast (GM4912, GM1935, GM3329) cell lines from infantile or adult PD exposed to mock treatment, L-GGB 50-100kD fraction, human placental GAA or rhGAA for 48 hours and assayed for GAA. All treatments were significant at p = ≤0.05.

Article Snippet: ERT with a recombinant human GAA (rhGAA) secreted by CHO cells (alglucosidase alfa/Myozyme/Lumizyme, Genzyme/Sanofi Corp.) infused every two weeks is the first approved therapy.

Techniques:

Journal: bioRxiv

Article Title: Preclinical studies with ground germinated barley (GGB) for oral enzyme replacement therapy (Oral-ERT) in Pompe disease knockout mice

doi: 10.1101/2023.12.27.573457

Figure Lengend Snippet: is a bar graph showing human normal and PD myoblast cells exposed to various amounts of L-GGB or L-GGB 50-100kD or rhGAA for 48 hours and assayed for GAA. We found an increased GAA to almost 50% of normal (mean+SD). All treatments were significant at p = <0.05.

Article Snippet: ERT with a recombinant human GAA (rhGAA) secreted by CHO cells (alglucosidase alfa/Myozyme/Lumizyme, Genzyme/Sanofi Corp.) infused every two weeks is the first approved therapy.

Techniques:

Journal: bioRxiv

Article Title: Preclinical studies with ground germinated barley (GGB) for oral enzyme replacement therapy (Oral-ERT) in Pompe disease knockout mice

doi: 10.1101/2023.12.27.573457

Figure Lengend Snippet:

Article Snippet: ERT with a recombinant human GAA (rhGAA) secreted by CHO cells (alglucosidase alfa/Myozyme/Lumizyme, Genzyme/Sanofi Corp.) infused every two weeks is the first approved therapy.

Techniques: Comparison

Journal: bioRxiv

Article Title: Preclinical studies with ground germinated barley (GGB) for oral enzyme replacement therapy (Oral-ERT) in Pompe disease knockout mice

doi: 10.1101/2023.12.27.573457

Figure Lengend Snippet: is a bar graph showing human normal and PD myoblast cells exposed to various amounts of L-GGB or L-GGB 50-100kD or rhGAA for 48 hours and assayed for GAA. We found an increased GAA to almost 50% of normal (mean+SD). All treatments were significant at p = <0.05.

Article Snippet: Mock treated GAA and normal myoblast cells were controls plus cells treated with equivalent amounts of a rhGAA (R&D Systems #8329-GH-025).

Techniques:

Journal: Biomolecules

Article Title: The Release of a Soluble Glycosylated Protein from Glycogen by Recombinant Lysosomal α-Glucosidase (rhGAA) In Vitro and Its Presence in Serum In Vivo

doi: 10.3390/biom10121613

Figure Lengend Snippet: ( A ) Incubation medium of Control 2 with rhGAA after 4 and 6 days, HCl extract of the same and 2N TFA hydrolyzate of same. ( B ) Incubation medium of Control 2 glycogen with rhGAA after 4 days: 2N TFA hydrolyzate and same incubated with rhGAA. ( C ) Incubation medium of Control 2 glycogen with rhGAA after 6 days: 2N TFA hydrolyzate and same incubated with rhGAA.

Article Snippet: Recombinant human GAA (rhGAA) was provided by Sanofi Genzyme, Framingham, MA, USA, which is the 110 kDa precursor which is converted to the mature form in the tissue in ERT.

Techniques: Incubation, Control

Journal: Biomolecules

Article Title: The Release of a Soluble Glycosylated Protein from Glycogen by Recombinant Lysosomal α-Glucosidase (rhGAA) In Vitro and Its Presence in Serum In Vivo

doi: 10.3390/biom10121613

Figure Lengend Snippet: ( A ) Extraction procedures for incubation medium from rhGAA degradation of Control 2 glycogen after no more glucose is released. HPAEC-PAD numbers 1–6 refer to chromatograms 1–6 in 2B. ( B ) Results of samples analysis at each of sic steps in 2A.

Article Snippet: Recombinant human GAA (rhGAA) was provided by Sanofi Genzyme, Framingham, MA, USA, which is the 110 kDa precursor which is converted to the mature form in the tissue in ERT.

Techniques: Extraction, Incubation, Control

Journal: Biomolecules

Article Title: The Release of a Soluble Glycosylated Protein from Glycogen by Recombinant Lysosomal α-Glucosidase (rhGAA) In Vitro and Its Presence in Serum In Vivo

doi: 10.3390/biom10121613

Figure Lengend Snippet: ( A ) Glycogen HCl extract showing maltooligosaccharides DP2-16 and clot HCl extract with array of maltooligosaccharides and the Unknown. ( B ) Serum, serum HCl extract, and serum HCl extract after rhGAA incubation.

Article Snippet: Recombinant human GAA (rhGAA) was provided by Sanofi Genzyme, Framingham, MA, USA, which is the 110 kDa precursor which is converted to the mature form in the tissue in ERT.

Techniques: Incubation

Journal: Biomolecules

Article Title: The Release of a Soluble Glycosylated Protein from Glycogen by Recombinant Lysosomal α-Glucosidase (rhGAA) In Vitro and Its Presence in Serum In Vivo

doi: 10.3390/biom10121613

Figure Lengend Snippet: ( A ) Clot HCl extract before and after degradation with rhGAA demonstrating the Unknown is not degraded. ( B ) HCl extract of Control 2 glycogen, HCl extract of clot following rhGAA degradation, and a mixture of equal parts of both extracts demonstrating one symmetrical peak.

Article Snippet: Recombinant human GAA (rhGAA) was provided by Sanofi Genzyme, Framingham, MA, USA, which is the 110 kDa precursor which is converted to the mature form in the tissue in ERT.

Techniques: Control