Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency
| dc.contributor.author | Gruntman, Alisha | |
| dc.contributor.author | Flotte, Terence R. | |
| dc.date | 2022-08-11T08:10:13.000 | |
| dc.date.accessioned | 2022-08-23T16:59:27Z | |
| dc.date.available | 2022-08-23T16:59:27Z | |
| dc.date.issued | 2017-07-28 | |
| dc.date.submitted | 2017-08-28 | |
| dc.identifier.citation | Methods Mol Biol. 2017;1639:267-275. doi: 10.1007/978-1-4939-7163-3_27. <a href="https://doi.org/10.1007/978-1-4939-7163-3_27">Link to article on publisher's site</a> | |
| dc.identifier.issn | 1064-3745 (Linking) | |
| dc.identifier.doi | 10.1007/978-1-4939-7163-3_27 | |
| dc.identifier.pmid | 28752467 | |
| dc.identifier.uri | http://hdl.handle.net/20.500.14038/43589 | |
| dc.description.abstract | This review seeks to give an overview of alpha-1 antitrypsin deficiency, including the different disease phenotypes that it encompasses. We then describe the different therapeutic endeavors that have been undertaken to address these different phenotypes. Lastly we discuss future potential therapeutics, such as genome editing, and how they may play a role in treating alpha-1 antitrypsin deficiency. | |
| dc.language.iso | en_US | |
| dc.relation | <a href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&list_uids=28752467&dopt=Abstract">Link to Article in PubMed</a> | |
| dc.relation.url | https://doi.org/10.1007/978-1-4939-7163-3_27 | |
| dc.subject | CRISPR/Cas9 | |
| dc.subject | Emphysema | |
| dc.subject | Genome editing | |
| dc.subject | Liver disease | |
| dc.subject | SERPINA1 | |
| dc.subject | Genetics and Genomics | |
| dc.subject | Molecular Biology | |
| dc.subject | Therapeutics | |
| dc.title | Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency | |
| dc.type | Book Chapter | |
| dc.source.booktitle | Methods in molecular biology (Clifton, N.J.) | |
| dc.source.volume | 1639 | |
| dc.identifier.legacycoverpage | https://escholarship.umassmed.edu/peds_pp/159 | |
| dc.identifier.contextkey | 10667370 | |
| html.description.abstract | <p>This review seeks to give an overview of alpha-1 antitrypsin deficiency, including the different disease phenotypes that it encompasses. We then describe the different therapeutic endeavors that have been undertaken to address these different phenotypes. Lastly we discuss future potential therapeutics, such as genome editing, and how they may play a role in treating alpha-1 antitrypsin deficiency.</p> | |
| dc.identifier.submissionpath | peds_pp/159 | |
| dc.contributor.department | Department of Pediatrics, Division of Pulmonary and Allergy | |
| dc.contributor.department | Horae Gene Therapy Center | |
| dc.source.pages | 267-275 |