Please use this identifier to cite or link to this item:
https://doi.org/10.1038/s41434-022-00313-w
DC Field | Value | |
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dc.title | First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model | |
dc.contributor.author | Sia, Kian Chuan | |
dc.contributor.author | Gan, Shu Uin | |
dc.contributor.author | Rodhi, Siti Humairah Mohd | |
dc.contributor.author | Fu, Zhen Ying | |
dc.contributor.author | Kopchick, John J | |
dc.contributor.author | Waters, Michael J | |
dc.contributor.author | Lee, Kok Onn | |
dc.date.accessioned | 2022-11-14T09:33:02Z | |
dc.date.available | 2022-11-14T09:33:02Z | |
dc.date.issued | 2022-02-01 | |
dc.identifier.citation | Sia, Kian Chuan, Gan, Shu Uin, Rodhi, Siti Humairah Mohd, Fu, Zhen Ying, Kopchick, John J, Waters, Michael J, Lee, Kok Onn (2022-02-01). First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model. GENE THERAPY 29 (6) : 346-356. ScholarBank@NUS Repository. https://doi.org/10.1038/s41434-022-00313-w | |
dc.identifier.issn | 0969-7128 | |
dc.identifier.issn | 1476-5462 | |
dc.identifier.uri | https://scholarbank.nus.edu.sg/handle/10635/234498 | |
dc.description.abstract | The only treatment tested for growth hormone receptor (GHR) defective Laron Syndrome (LS) is injections of recombinant insulin-like-growth factor 1 (rhIGF1). The response is suboptimal and associated with progressive obesity. In this study, we treated 4–5-week-old Laron dwarf mice (GHR−/−) with an adeno-associated virus expressing murine GHR (AAV-GHR) injection at a dose of 4 × 1010 vector genome per mouse. Serum growth hormone (GH) levels decreased, and GH-responsive IGF1, IGF binding protein 3 (IGFBP3) and acid labile subunit (ALS) increased. There was a significant but limited increase in body weight and length, similar to the response to rhIGF1 treatment in LS patients. All the major organs increased in weight except the brain. Our study is the first to use gene therapy to treat GH-receptor deficiency. We propose that gene therapy with AAV-GHR may eventually be useful for the treatment of human LS. | |
dc.language.iso | en | |
dc.publisher | SPRINGERNATURE | |
dc.source | Elements | |
dc.subject | Science & Technology | |
dc.subject | Life Sciences & Biomedicine | |
dc.subject | Biochemistry & Molecular Biology | |
dc.subject | Biotechnology & Applied Microbiology | |
dc.subject | Genetics & Heredity | |
dc.subject | Medicine, Research & Experimental | |
dc.subject | Research & Experimental Medicine | |
dc.subject | ADENOASSOCIATED VIRUS VECTOR | |
dc.subject | LONG-TERM TREATMENT | |
dc.subject | LARON-SYNDROME | |
dc.subject | FACTOR-I | |
dc.subject | IGF-I | |
dc.subject | INSENSITIVITY SYNDROME | |
dc.subject | CHILDREN | |
dc.subject | LIVER | |
dc.subject | DISRUPTION | |
dc.subject | DEFICIENCY | |
dc.type | Article | |
dc.date.updated | 2022-11-14T02:06:31Z | |
dc.contributor.department | MEDICINE | |
dc.contributor.department | SURGERY | |
dc.description.doi | 10.1038/s41434-022-00313-w | |
dc.description.sourcetitle | GENE THERAPY | |
dc.description.volume | 29 | |
dc.description.issue | 6 | |
dc.description.page | 346-356 | |
dc.published.state | Published | |
Appears in Collections: | Staff Publications Elements |
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Sia et al. - 2022 - First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model.pdf | Published version | 2.54 MB | Adobe PDF | OPEN | Published | View/Download |
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