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Their heterogeneity, diversity, and nature pose daunting clinical challenges for both management and treatment. In this review, we discuss recent advances in clinical applications of gene therapy for rare diseases, focusing on a variety of viral and non\u2010viral strategies. The use of adeno\u2010associated virus (AAV) vectors is discussed in the context of Luxturna, licenced for the treatment of\n                    <jats:italic>RPE65<\/jats:italic>\n                    deficiency in the retinal epithelium. Imlygic, a herpes virus vector licenced for the treatment of refractory metastatic melanoma, will be an example of oncolytic vectors developed against rare cancers. Yescarta and Kymriah will showcase the use of retrovirus and lentivirus vectors in the autologous ex vivo production of chimeric antigen receptor T cells (CAR\u2010T), licenced for the treatment of refractory leukaemias and lymphomas. Similar retroviral and lentiviral technology can be applied to autologous haematopoietic stem cells, exemplified by Strimvelis and Zynteglo, licenced treatments for adenosine deaminase\u2010severe combined immunodeficiency (ADA\u2010SCID) and \u03b2\u2010thalassaemia respectively. Antisense oligonucleotide technologies will be highlighted through Onpattro and Tegsedi, RNA interference drugs licenced for familial transthyretin (TTR) amyloidosis, and Spinraza, a splice\u2010switching treatment for spinal muscular atrophy (SMA). An initial comparison of the effectiveness of AAV and oligonucleotide therapies in SMA is possible with Zolgensma, an AAV serotype 9 vector, and Spinraza. Through these examples of marketed gene therapies and gene cell therapies, we will discuss the expanding applications of such novel technologies to previously intractable rare diseases.\n                  <\/jats:p>","DOI":"10.1111\/iep.12478","type":"journal-article","created":{"date-parts":[[2023,5,13]],"date-time":"2023-05-13T03:04:39Z","timestamp":1683947079000},"page":"154-176","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":60,"title":["Clinical applications of gene therapy for rare diseases: A review"],"prefix":"10.1111","volume":"104","author":[{"given":"Ioannis","family":"Papaioannou","sequence":"first","affiliation":[{"name":"Division of Medicine University College London  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5281-4001","authenticated-orcid":false,"given":"James S.","family":"Owen","sequence":"additional","affiliation":[{"name":"Division of Medicine University College London  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7113-1938","authenticated-orcid":false,"given":"Rafael J.","family":"Y\u00e1\u00f1ez\u2010Mu\u00f1oz","sequence":"additional","affiliation":[{"name":"AGCTlab.org Centre of Gene and Cell Therapy Centre for Biomedical Sciences Department of Biological Sciences School of Life Sciences and the Environment Royal Holloway University of London  Egham UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,5,13]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/d41573-019-00180-y"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41573-019-0049-9"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.2174\/0929867324666170511111803"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41431-019-0508-0"},{"key":"e_1_2_9_6_1","first-page":"151","article-title":"Autosomal dominant congenital macroglossia: further delineation of the syndrome","volume":"5","author":"Reynoso MC","year":"1994","journal-title":"Genet Couns"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1007\/BF00282095"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41582-020-0389-4"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrdp.2015.5"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrdp.2015.61"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2009-06-189209"},{"key":"e_1_2_9_12_1","unstructured":"Rare diseases: understanding this Public Health Priority. 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