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dc.contributor.authorGruntman, Alisha M
dc.contributor.authorFlotte, Terence R.
dc.date2022-08-11T08:10:13.000
dc.date.accessioned2022-08-23T16:59:27Z
dc.date.available2022-08-23T16:59:27Z
dc.date.issued2017-07-28
dc.date.submitted2017-08-28
dc.identifier.citationMethods 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.issn1064-3745 (Linking)
dc.identifier.doi10.1007/978-1-4939-7163-3_27
dc.identifier.pmid28752467
dc.identifier.urihttp://hdl.handle.net/20.500.14038/43589
dc.description.abstractThis 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.isoen_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.urlhttps://doi.org/10.1007/978-1-4939-7163-3_27
dc.subjectCRISPR/Cas9
dc.subjectEmphysema
dc.subjectGenome editing
dc.subjectLiver disease
dc.subjectSERPINA1
dc.subjectGenetics and Genomics
dc.subjectMolecular Biology
dc.subjectTherapeutics
dc.titleTherapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency
dc.typeBook Chapter
dc.source.booktitleMethods in molecular biology (Clifton, N.J.)
dc.source.volume1639
dc.identifier.legacycoverpagehttps://escholarship.umassmed.edu/peds_pp/159
dc.identifier.contextkey10667370
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.submissionpathpeds_pp/159
dc.contributor.departmentDepartment of Pediatrics, Division of Pulmonary and Allergy
dc.contributor.departmentHorae Gene Therapy Center
dc.source.pages267-275


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