Promise of gene therapy for congenital neurologic disease due to GPI deficiency
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2024-06-01
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| Series: | Molecular Therapy: Methods & Clinical Development |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050124000561 |
| _version_ | 1797242969824165888 |
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| author | Daria V. Babushok Denise E. Sabatino |
| author_facet | Daria V. Babushok Denise E. Sabatino |
| author_sort | Daria V. Babushok |
| collection | DOAJ |
| first_indexed | 2024-04-24T18:47:40Z |
| format | Article |
| id | doaj.art-a7208f3a334240609a93314c889a9105 |
| institution | Directory Open Access Journal |
| issn | 2329-0501 |
| language | English |
| last_indexed | 2024-04-24T18:47:40Z |
| publishDate | 2024-06-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Molecular Therapy: Methods & Clinical Development |
| spelling | doaj.art-a7208f3a334240609a93314c889a91052024-03-27T04:52:19ZengElsevierMolecular Therapy: Methods & Clinical Development2329-05012024-06-01322101240Promise of gene therapy for congenital neurologic disease due to GPI deficiencyDaria V. Babushok0Denise E. Sabatino1Division of Hematology-Oncology, Department of Medicine, University of Pennsylvania, 3400 Civic Center Boulevard, Philadelphia, PA 19104, USA; Comprehensive Bone Marrow Failure Center, Department of Pediatrics, Children’s Hospital of Philadelphia, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USA; Corresponding author: Daria Babushok, Division of Hematology-Oncology, Department of Medicine, University of Pennsylvania, BRB II/III, Room 808, 421 Curie Boulevard, Philadelphia, PA 19104, USA.The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA, USA; Division of Hematology, Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAhttp://www.sciencedirect.com/science/article/pii/S2329050124000561 |
| spellingShingle | Daria V. Babushok Denise E. Sabatino Promise of gene therapy for congenital neurologic disease due to GPI deficiency Molecular Therapy: Methods & Clinical Development |
| title | Promise of gene therapy for congenital neurologic disease due to GPI deficiency |
| title_full | Promise of gene therapy for congenital neurologic disease due to GPI deficiency |
| title_fullStr | Promise of gene therapy for congenital neurologic disease due to GPI deficiency |
| title_full_unstemmed | Promise of gene therapy for congenital neurologic disease due to GPI deficiency |
| title_short | Promise of gene therapy for congenital neurologic disease due to GPI deficiency |
| title_sort | promise of gene therapy for congenital neurologic disease due to gpi deficiency |
| url | http://www.sciencedirect.com/science/article/pii/S2329050124000561 |
| work_keys_str_mv | AT dariavbabushok promiseofgenetherapyforcongenitalneurologicdiseaseduetogpideficiency AT deniseesabatino promiseofgenetherapyforcongenitalneurologicdiseaseduetogpideficiency |