Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease
Pompe disease is a rare genetic neuromuscular disorder caused by acid α-glucosidase (GAA) deficiency resulting in lysosomal glycogen accumulation and progressive myopathy. Enzyme replacement therapy, the current standard of care, penetrates poorly into the skeletal muscles and the peripheral and cen...
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| Published in: | Molecular therapy. Methods & clinical development Vol. 27; pp. 464 - 487 |
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| Main Authors: | , , , , , , , , , , , , , , |
| Format: | Journal Article |
| Language: | English |
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Elsevier Inc
08-12-2022
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| Abstract | Pompe disease is a rare genetic neuromuscular disorder caused by acid α-glucosidase (GAA) deficiency resulting in lysosomal glycogen accumulation and progressive myopathy. Enzyme replacement therapy, the current standard of care, penetrates poorly into the skeletal muscles and the peripheral and central nervous system (CNS), risks recombinant enzyme immunogenicity, and requires high doses and frequent infusions. Lentiviral vector-mediated hematopoietic stem and progenitor cell (HSPC) gene therapy was investigated in a Pompe mouse model using a clinically relevant promoter driving nine engineered GAA coding sequences incorporating distinct peptide tags and codon optimizations. Vectors solely including glycosylation-independent lysosomal targeting tags enhanced secretion and improved reduction of glycogen, myofiber, and CNS vacuolation in key tissues, although GAA enzyme activity and protein was consistently lower compared with native GAA. Genetically modified microglial cells in brains were detected at low levels but provided robust phenotypic correction. Furthermore, an amino acid substitution introduced in the tag reduced insulin receptor-mediated signaling with no evidence of an effect on blood glucose levels in Pompe mice. This study demonstrated the therapeutic potential of lentiviral HSPC gene therapy exploiting optimized GAA tagged coding sequences to reverse Pompe disease pathology in a preclinical mouse model, providing promising vector candidates for further investigation.
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| AbstractList | Pompe disease is a rare genetic neuromuscular disorder caused by acid α-glucosidase (GAA) deficiency resulting in lysosomal glycogen accumulation and progressive myopathy. Enzyme replacement therapy, the current standard of care, penetrates poorly into the skeletal muscles and the peripheral and central nervous system (CNS), risks recombinant enzyme immunogenicity, and requires high doses and frequent infusions. Lentiviral vector-mediated hematopoietic stem and progenitor cell (HSPC) gene therapy was investigated in a Pompe mouse model using a clinically relevant promoter driving nine engineered GAA coding sequences incorporating distinct peptide tags and codon optimizations. Vectors solely including glycosylation-independent lysosomal targeting tags enhanced secretion and improved reduction of glycogen, myofiber, and CNS vacuolation in key tissues, although GAA enzyme activity and protein was consistently lower compared with native GAA. Genetically modified microglial cells in brains were detected at low levels but provided robust phenotypic correction. Furthermore, an amino acid substitution introduced in the tag reduced insulin receptor-mediated signaling with no evidence of an effect on blood glucose levels in Pompe mice. This study demonstrated the therapeutic potential of lentiviral HSPC gene therapy exploiting optimized GAA tagged coding sequences to reverse Pompe disease pathology in a preclinical mouse model, providing promising vector candidates for further investigation. Pompe disease is a rare genetic neuromuscular disorder caused by acid α-glucosidase (GAA) deficiency resulting in lysosomal glycogen accumulation and progressive myopathy. Enzyme replacement therapy, the current standard of care, penetrates poorly into the skeletal muscles and the peripheral and central nervous system (CNS), risks recombinant enzyme immunogenicity, and requires high doses and frequent infusions. Lentiviral vector-mediated hematopoietic stem and progenitor cell (HSPC) gene therapy was investigated in a Pompe mouse model using a clinically relevant promoter driving nine engineered GAA coding sequences incorporating distinct peptide tags and codon optimizations. Vectors solely including glycosylation-independent lysosomal targeting tags enhanced secretion and improved reduction of glycogen, myofiber, and CNS vacuolation in key tissues, although GAA enzyme activity and protein was consistently lower compared with native GAA. Genetically modified microglial cells in brains were detected at low levels but provided robust phenotypic correction. Furthermore, an amino acid substitution introduced in the tag reduced insulin receptor-mediated signaling with no evidence of an effect on blood glucose levels in Pompe mice. This study demonstrated the therapeutic potential of lentiviral HSPC gene therapy exploiting optimized GAA tagged coding sequences to reverse Pompe disease pathology in a preclinical mouse model, providing promising vector candidates for further investigation. [Display omitted] |
| Author | Guda, Swaroopa Pfeifer, Richard Harper, Claudia Schambach, Axel Schindler, Jeffrey W. Dogan, Yildirim van Til, Niek P. Oborski, Christine Maiwald, Tim Mason, Chris Barese, Cecilia N. Yoon, John K. Jacobs, Mary E. Clarke, Diana L. Unnisa, Zeenath |
| Author_xml | – sequence: 1 givenname: Yildirim surname: Dogan fullname: Dogan, Yildirim organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 2 givenname: Cecilia N. surname: Barese fullname: Barese, Cecilia N. organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 3 givenname: Jeffrey W. surname: Schindler fullname: Schindler, Jeffrey W. organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 4 givenname: John K. surname: Yoon fullname: Yoon, John K. organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 5 givenname: Zeenath surname: Unnisa fullname: Unnisa, Zeenath organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 6 givenname: Swaroopa surname: Guda fullname: Guda, Swaroopa organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 7 givenname: Mary E. surname: Jacobs fullname: Jacobs, Mary E. organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 8 givenname: Christine surname: Oborski fullname: Oborski, Christine organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 9 givenname: Tim surname: Maiwald fullname: Maiwald, Tim organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 10 givenname: Diana L. surname: Clarke fullname: Clarke, Diana L. organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 11 givenname: Axel surname: Schambach fullname: Schambach, Axel organization: Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Straße 1, 30625 Hannover, Germany – sequence: 12 givenname: Richard surname: Pfeifer fullname: Pfeifer, Richard organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 13 givenname: Claudia surname: Harper fullname: Harper, Claudia organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 14 givenname: Chris surname: Mason fullname: Mason, Chris email: chris.mason@ucl.ac.uk organization: AVROBIO, Inc., Cambridge, MA 02139, USA – sequence: 15 givenname: Niek P. surname: van Til fullname: van Til, Niek P. email: n.p.vantil@amsterdamumc.nl organization: AVROBIO, Inc., Cambridge, MA 02139, USA |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/36419467$$D View this record in MEDLINE/PubMed |
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| Keywords | Pompe disease hematopoietic stem and progenitor cells glycosylation-independent lysosomal targeting lentiviral vector tag technology |
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