{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T12:16:34Z","timestamp":1791288994732,"version":"4.3.0"},"reference-count":197,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2023,5,2]],"date-time":"2023-05-02T00:00:00Z","timestamp":1682985600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2023,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic diseases caused by functional defects in specific lysosomal proteins. The lysosome is a cellular organelle that plays a critical role in catabolism of waste products and recycling of macromolecules in the body. Disruption to the normal function of the lysosome can result in the toxic accumulation of storage products, often leading to irreparable cellular damage and organ dysfunction followed by premature death. The majority of LSDs have no curative treatment, with many clinical subtypes presenting in early infancy and childhood. Over two\u2010thirds of LSDs present with progressive neurodegeneration, often in combination with other debilitating peripheral symptoms. Consequently, there is a pressing unmet clinical need to develop new therapeutic interventions to treat these conditions. The blood\u2013brain barrier is a crucial hurdle that needs to be overcome in order to effectively treat the central nervous system (CNS), adding considerable complexity to therapeutic design and delivery. Enzyme replacement therapy (ERT) treatments aimed at either direct injection into the brain, or using blood\u2013brain barrier constructs are discussed, alongside more conventional substrate reduction and other drug\u2010related therapies. Other promising strategies developed in recent years, include gene therapy technologies specifically tailored for more effectively targeting treatment to the CNS. Here, we discuss the most recent advances in CNS\u2010targeted treatments for neurological LSDs with a particular emphasis on gene therapy\u2010based modalities, such as Adeno\u2010Associated Virus and haematopoietic stem cell gene therapy approaches that encouragingly, at the time of writing are being evaluated in LSD clinical trials in increasing numbers. If safety, efficacy and improved quality of life can be demonstrated, these therapies have the potential to be the new standard of care treatments for LSD patients.<\/jats:p>","DOI":"10.1002\/jimd.12615","type":"journal-article","created":{"date-parts":[[2023,4,20]],"date-time":"2023-04-20T07:27:01Z","timestamp":1681975621000},"page":"874-905","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":57,"title":["Advances in therapies for neurological lysosomal storage disorders"],"prefix":"10.1002","volume":"46","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7373-4951","authenticated-orcid":false,"given":"S.","family":"Ellison","sequence":"first","affiliation":[{"name":"Division of Cell Matrix Biology and Regenerative Medicine University of Manchester Manchester United\u00a0Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7324-9710","authenticated-orcid":false,"given":"H.","family":"Parker","sequence":"additional","affiliation":[{"name":"Division of Immunology, Immunity to Infection and Respiratory Medicine University of Manchester Manchester United\u00a0Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9708-1112","authenticated-orcid":false,"given":"B.","family":"Bigger","sequence":"additional","affiliation":[{"name":"Division of Cell Matrix Biology and Regenerative Medicine University of Manchester Manchester United\u00a0Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,5,2]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.202102001"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.21037\/atm.2018.11.39"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s004399900075"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.281.3.249"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.lanwpc.2021.100344"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2021.05.004"},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2022.908657"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1080\/19490976.2022.2027852"},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm10143026"},{"key":"e_1_2_13_11_1","doi-asserted-by":"publisher","DOI":"10.1023\/a:1012463605992"},{"key":"e_1_2_13_12_1","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12431"},{"key":"e_1_2_13_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/ans.17430"},{"key":"e_1_2_13_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bone.2020.115729"},{"key":"e_1_2_13_15_1","volume-title":"Lysosomal Storage Disorders: A Practical Guide","year":"2022"},{"key":"e_1_2_13_16_1","doi-asserted-by":"publisher","DOI":"10.3389\/fnins.2015.00417"},{"key":"e_1_2_13_17_1","doi-asserted-by":"publisher","DOI":"10.1080\/15548627.2015.1046671"},{"key":"e_1_2_13_18_1","doi-asserted-by":"publisher","DOI":"10.4161\/auto.19469"},{"key":"e_1_2_13_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2018.08.003"},{"key":"e_1_2_13_20_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms23041999"},{"key":"e_1_2_13_21_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm9020396"},{"key":"e_1_2_13_22_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0035787"},{"key":"e_1_2_13_23_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2012.82"},{"key":"e_1_2_13_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/jnc.14632"},{"key":"e_1_2_13_25_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awx311"},{"key":"e_1_2_13_26_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2013.141"},{"key":"e_1_2_13_27_1","doi-asserted-by":"publisher","DOI":"10.1101\/cshperspect.a020628"},{"key":"e_1_2_13_28_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrn3908"},{"key":"e_1_2_13_29_1","doi-asserted-by":"publisher","DOI":"10.1038\/ncpneuro0355"},{"key":"e_1_2_13_30_1","doi-asserted-by":"publisher","DOI":"10.4103\/1673\u20105374.265541"},{"key":"e_1_2_13_31_1","doi-asserted-by":"publisher","DOI":"10.1111\/tra.12714"},{"key":"e_1_2_13_32_1","doi-asserted-by":"publisher","DOI":"10.3233\/TRD\u2010160005"},{"key":"e_1_2_13_33_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11011\u2010017\u20100009\u20101"},{"key":"e_1_2_13_34_1","doi-asserted-by":"publisher","DOI":"10.1002\/glia.23598"},{"key":"e_1_2_13_35_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.beem.2014.10.001"},{"key":"e_1_2_13_36_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.1233158"},{"key":"e_1_2_13_37_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010018\u20100872\u20109"},{"key":"e_1_2_13_38_1","doi-asserted-by":"publisher","DOI":"10.1002\/jnr.23873"},{"key":"e_1_2_13_39_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073813493664"},{"key":"e_1_2_13_40_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073813493665"},{"key":"e_1_2_13_41_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2009.07.030"},{"key":"e_1_2_13_42_1","doi-asserted-by":"publisher","DOI":"10.3389\/fnagi.2019.00373"},{"key":"e_1_2_13_43_1","doi-asserted-by":"publisher","DOI":"10.4155\/tde.15.48"},{"key":"e_1_2_13_44_1","doi-asserted-by":"publisher","DOI":"10.1208\/s12248\u2010008\u20109055\u20102"},{"key":"e_1_2_13_45_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0016601"},{"key":"e_1_2_13_46_1","doi-asserted-by":"publisher","DOI":"10.1186\/1471\u20102377\u201013\u2010174"},{"key":"e_1_2_13_47_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.addr.2017.05.004"},{"key":"e_1_2_13_48_1","doi-asserted-by":"publisher","DOI":"10.1016\/s0268\u2010960x(98)90023\u20106"},{"key":"e_1_2_13_49_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41598\u2010019\u201050595\u20101"},{"key":"e_1_2_13_50_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2016.08.033"},{"key":"e_1_2_13_51_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2008.07.004"},{"key":"e_1_2_13_52_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2006\u20102156"},{"key":"e_1_2_13_53_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0184065"},{"key":"e_1_2_13_54_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2018.04.013"},{"key":"e_1_2_13_55_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010017\u20100712\u20103"},{"key":"e_1_2_13_56_1","doi-asserted-by":"publisher","DOI":"10.1038\/gim.2015.36"},{"key":"e_1_2_13_57_1","doi-asserted-by":"publisher","DOI":"10.1097\/01.gim.0000232477.37660.fb"},{"key":"e_1_2_13_58_1","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12028"},{"key":"e_1_2_13_59_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010017\u20100634\u20100"},{"key":"e_1_2_13_60_1","doi-asserted-by":"publisher","DOI":"10.1038\/pr.2015.169"},{"key":"e_1_2_13_61_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2020.02.009"},{"key":"e_1_2_13_62_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgmr.2019.100490"},{"key":"e_1_2_13_63_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2015.05.012"},{"key":"e_1_2_13_64_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bcmd.2016.07.002"},{"key":"e_1_2_13_65_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41436\u2010019\u20100738\u20100"},{"key":"e_1_2_13_66_1","doi-asserted-by":"publisher","DOI":"10.2174\/156652311794940791"},{"key":"e_1_2_13_67_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gim.2022.04.002"},{"key":"e_1_2_13_68_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2019.11.007"},{"key":"e_1_2_13_69_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2021.07.001"},{"key":"e_1_2_13_70_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2018.10.006"},{"key":"e_1_2_13_71_1","unstructured":"Clinigen press release 2021.https:\/\/www.clinigengroup.com\/news\/news\u2010container\/2021\/clinigen\u2010receives\u2010marketing\u2010approval\u2010for\u2010hunterase\u2010idursulfase\u2010beta\u2010icv\u2010in\u2010japan\/"},{"key":"e_1_2_13_72_1","unstructured":"FDA press release 2017. FDA approves first treatment for a form of Batten disease.https:\/\/www.fda.gov\/news\u2010events\/press\u2010announcements\/fda\u2010approves\u2010first\u2010treatment\u2010form\u2010batten\u2010disease"},{"key":"e_1_2_13_73_1","first-page":"549","article-title":"Development of nanoparticle\u2010bound arylsulfatase B for enzyme replacement therapy of mucopolysaccharidosis VI","volume":"68","author":"Muhlstein A","year":"2013","journal-title":"Pharmazie"},{"key":"e_1_2_13_74_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2013.267"},{"key":"e_1_2_13_75_1","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.4785\u201013.2014"},{"key":"e_1_2_13_76_1","doi-asserted-by":"publisher","DOI":"10.1002\/bit.25289"},{"key":"e_1_2_13_77_1","doi-asserted-by":"publisher","DOI":"10.1021\/mp500258p"},{"key":"e_1_2_13_78_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1416660111"},{"key":"e_1_2_13_79_1","doi-asserted-by":"publisher","DOI":"10.1177\/0271678X15614589"},{"key":"e_1_2_13_80_1","doi-asserted-by":"publisher","DOI":"10.1042\/BJ20130845"},{"key":"e_1_2_13_81_1","doi-asserted-by":"publisher","DOI":"10.1021\/acs.molpharmaceut.6b00037"},{"key":"e_1_2_13_82_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms20082014"},{"key":"e_1_2_13_83_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2019.05.008"},{"key":"e_1_2_13_84_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010018\u20100849\u20108"},{"key":"e_1_2_13_85_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms21020400"},{"key":"e_1_2_13_86_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2018.12.005"},{"key":"e_1_2_13_87_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgmr.2019.100510"},{"key":"e_1_2_13_88_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2020.01.001"},{"key":"e_1_2_13_89_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.beem.2015.01.006"},{"key":"e_1_2_13_90_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2019.02.007"},{"key":"e_1_2_13_91_1","doi-asserted-by":"publisher","DOI":"10.1038\/mtm.2015.53"},{"key":"e_1_2_13_92_1","doi-asserted-by":"publisher","DOI":"10.1002\/emmm.200900036"},{"key":"e_1_2_13_93_1","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2017.00448"},{"key":"e_1_2_13_94_1","doi-asserted-by":"publisher","DOI":"10.1517\/14728214.2010.498580"},{"key":"e_1_2_13_95_1","doi-asserted-by":"publisher","DOI":"10.2183\/pjab.90.145"},{"key":"e_1_2_13_96_1","doi-asserted-by":"publisher","DOI":"10.15252\/emmm.201708730"},{"key":"e_1_2_13_97_1","doi-asserted-by":"publisher","DOI":"10.1023\/b:boli.0000042959.44318.7c"},{"key":"e_1_2_13_98_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102010\u201003\u2010273151"},{"key":"e_1_2_13_99_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(07)70194-1"},{"key":"e_1_2_13_100_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140\u20106736(00)02161\u20109"},{"key":"e_1_2_13_101_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41436\u2010019\u20100451\u2010z"},{"key":"e_1_2_13_102_1","doi-asserted-by":"publisher","DOI":"10.1039\/c6cc01564f"},{"key":"e_1_2_13_103_1","doi-asserted-by":"publisher","DOI":"10.1136\/jmedgenet\u20102019\u2010106132"},{"key":"e_1_2_13_104_1","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.292"},{"key":"e_1_2_13_105_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M609304200"},{"key":"e_1_2_13_106_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010015\u20100260\u20107"},{"key":"e_1_2_13_107_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2009.008078"},{"key":"e_1_2_13_108_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010020\u201001363\u20102"},{"key":"e_1_2_13_109_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm11216265"},{"key":"e_1_2_13_110_1","unstructured":"National Gaucher Foundation.Substrate Reduction Therapy.https:\/\/www.gaucherdisease.org\/gaucher-diagnosis-treatment\/treatment\/substrate-reduction\/"},{"key":"e_1_2_13_111_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2012.04.005"},{"key":"e_1_2_13_112_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010018\u20100785\u20107"},{"key":"e_1_2_13_113_1","doi-asserted-by":"publisher","DOI":"10.1002\/mgg3.1371"},{"key":"e_1_2_13_114_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0014192"},{"key":"e_1_2_13_115_1","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12407"},{"key":"e_1_2_13_116_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2017.03.025"},{"key":"e_1_2_13_117_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2019.217"},{"key":"e_1_2_13_118_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND\u2010190426"},{"key":"e_1_2_13_119_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2022.09.015"},{"key":"e_1_2_13_120_1","doi-asserted-by":"publisher","DOI":"10.3390\/biomedicines10040746"},{"key":"e_1_2_13_121_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.molmed.2021.03.010"},{"key":"e_1_2_13_122_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2017.07.004"},{"key":"e_1_2_13_123_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2012.029"},{"key":"e_1_2_13_124_1","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2020.00670"},{"key":"e_1_2_13_125_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2019.12.010"},{"key":"e_1_2_13_126_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41593\u2010021\u201000969\u20104"},{"key":"e_1_2_13_127_1","doi-asserted-by":"publisher","DOI":"10.3389\/fnmol.2016.00116"},{"key":"e_1_2_13_128_1","doi-asserted-by":"publisher","DOI":"10.3389\/fnins.2021.747726"},{"key":"e_1_2_13_129_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2019.10.009"},{"key":"e_1_2_13_130_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2019.008"},{"key":"e_1_2_13_131_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2019.012"},{"key":"e_1_2_13_132_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2018.189"},{"key":"e_1_2_13_133_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2018.06.003"},{"key":"e_1_2_13_134_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2017.026"},{"key":"e_1_2_13_135_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.trsl.2022.05.002"},{"key":"e_1_2_13_136_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2021.245"},{"key":"e_1_2_13_137_1","doi-asserted-by":"publisher","DOI":"10.1172\/jci146286"},{"key":"e_1_2_13_138_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awy126"},{"key":"e_1_2_13_139_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2019.12.001"},{"key":"e_1_2_13_140_1","doi-asserted-by":"publisher","DOI":"10.1007\/978\u20103\u2010030\u201099166\u20101_6"},{"key":"e_1_2_13_141_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2019.08.009"},{"key":"e_1_2_13_142_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2016.184"},{"key":"e_1_2_13_143_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgmr.2015.11.006"},{"key":"e_1_2_13_144_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddv168"},{"key":"e_1_2_13_145_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gene.2015.06.049"},{"key":"e_1_2_13_146_1","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aac6191"},{"key":"e_1_2_13_147_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2015.05.003"},{"key":"e_1_2_13_148_1","doi-asserted-by":"publisher","DOI":"10.1089\/humc.2013.208"},{"key":"e_1_2_13_149_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41591\u2010021\u201001664\u20104"},{"key":"e_1_2_13_150_1","doi-asserted-by":"publisher","DOI":"10.3389\/fmolb.2021.624988"},{"key":"e_1_2_13_151_1","doi-asserted-by":"publisher","DOI":"10.3389\/fmolb.2022.965089"},{"key":"e_1_2_13_152_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2013.238"},{"key":"e_1_2_13_153_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(17)30169\u20102"},{"key":"e_1_2_13_154_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2018.12.283"},{"key":"e_1_2_13_155_1","unstructured":"Lysogene.Lysogene provides updates and topline results from phase 2\/3 AAVance gene therapy clinical study.2022https:\/\/www.lysogene.com\/lysogene-provides-updates-and-topline-results-from-phase-2-3-aavance-gene-therapy-clinical-study\/"},{"key":"e_1_2_13_156_1","unstructured":"Lysogene.Lysogene announces termination of license agreement with Sarepta for LYS\u2010SAF302 program regaining global commercial rights.https:\/\/www.lysogene.com\/lysogene\u2010announces\u2010termination\u2010of\u2010license\u2010agreement\u2010with\u2010sarepta\u2010for\u2010lys\u2010saf302\u2010program\u2010regaining\u2010global\u2010commercial\u2010rights\/2022."},{"key":"e_1_2_13_157_1","unstructured":"Sanfilippo news.Trial of ABO\u2010102 Gene Therapy Ends Citing No Neurocognitive Benefits.2022.https:\/\/sanfilipponews.com\/news\/trial\u2010of\u2010abo\u2010102\u2010gne\u2010therapy\u2010ends\u2010citing\u2010no\u2010neurocognitive\u2010benefits\/"},{"key":"e_1_2_13_158_1","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1212\/WNL.98.18_supplement.213","article-title":"Updated results of Transpher a, a multicenter, single\u2010dose, phase 1\/2 clinical trial of ABO\u2010102 investigational gene therapy for Sanfilippo syndrome type a (mucopolysaccharidosis IIIA) (S39.008)","volume":"98","author":"Flanigan KM","year":"2022","journal-title":"Neurology"},{"key":"e_1_2_13_159_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2022.170"},{"key":"e_1_2_13_160_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2017.11.007"},{"key":"e_1_2_13_161_1","doi-asserted-by":"publisher","DOI":"10.1089\/humc.2017.109"},{"key":"e_1_2_13_162_1","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2021.658038"},{"key":"e_1_2_13_163_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41434\u2010022\u201000368\u20109"},{"key":"e_1_2_13_164_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010020\u201001644\u2010w"},{"key":"e_1_2_13_165_1","doi-asserted-by":"publisher","DOI":"10.1007\/8904_2015_521"},{"key":"e_1_2_13_166_1","doi-asserted-by":"publisher","DOI":"10.1016\/s0140\u20106736(81)91046\u20101"},{"key":"e_1_2_13_167_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102014\u201011\u2010608075"},{"key":"e_1_2_13_168_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41390\u2010019\u20100541\u20102"},{"key":"e_1_2_13_169_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2011.058644"},{"key":"e_1_2_13_170_1","doi-asserted-by":"publisher","DOI":"10.15252\/emmm.201707573"},{"key":"e_1_2_13_171_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2013.265"},{"key":"e_1_2_13_172_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102017\u201004\u2010777136"},{"key":"e_1_2_13_173_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2015.3253"},{"key":"e_1_2_13_174_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI90367"},{"key":"e_1_2_13_175_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41591\u2010019\u20100735\u20105"},{"key":"e_1_2_13_176_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI28873"},{"key":"e_1_2_13_177_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2016.05.006"},{"key":"e_1_2_13_178_1","doi-asserted-by":"publisher","DOI":"10.1023\/a:1005526628598"},{"key":"e_1_2_13_179_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2019.04.001"},{"key":"e_1_2_13_180_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102020\u2010141762"},{"key":"e_1_2_13_181_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010017\u20100675\u20104"},{"key":"e_1_2_13_182_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2016.068"},{"key":"e_1_2_13_183_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2106596"},{"key":"e_1_2_13_184_1","doi-asserted-by":"publisher","DOI":"10.1002\/path.5078"},{"key":"e_1_2_13_185_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2014.152"},{"key":"e_1_2_13_186_1","first-page":"207","article-title":"Novel approaches and mechanisms in hematopoietic stem cell gene therapy","volume":"17","author":"Bigger BW","year":"2014","journal-title":"Discov Med"},{"key":"e_1_2_13_187_1","first-page":"179","article-title":"Bone marrow gene transfer in three patients with adenosine deaminase deficiency","volume":"3","author":"Hoogerbrugge PM","year":"1996","journal-title":"Gene Ther"},{"key":"e_1_2_13_188_1","doi-asserted-by":"publisher","DOI":"10.1038\/nm0798\u2010775"},{"key":"e_1_2_13_189_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.94.22.12133"},{"key":"e_1_2_13_190_1","first-page":"383","article-title":"Gene therapy for leukocyte adhesion deficiency","volume":"2","author":"Bauer TR","year":"2000","journal-title":"Curr Opin Mol Ther"},{"key":"e_1_2_13_191_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.17269"},{"key":"e_1_2_13_192_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2027675"},{"key":"e_1_2_13_193_1","doi-asserted-by":"publisher","DOI":"10.3390\/diseases7030047"},{"key":"e_1_2_13_194_1","doi-asserted-by":"publisher","DOI":"10.1016\/bs.pmbts.2021.01.026"},{"key":"e_1_2_13_195_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2018.03.002"},{"key":"e_1_2_13_196_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2018.10.018"},{"key":"e_1_2_13_197_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41434\u2010019\u20100113\u20104"},{"key":"e_1_2_13_198_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2019.05.014"}],"container-title":["Journal of Inherited Metabolic Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jimd.12615","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,12,11]],"date-time":"2023-12-11T01:34:44Z","timestamp":1702258484000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/jimd.12615"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,5,2]]},"references-count":197,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2023,9]]}},"alternative-id":["10.1002\/jimd.12615"],"URL":"https:\/\/doi.org\/10.1002\/jimd.12615","archive":["Portico"],"relation":{},"ISSN":["0141-8955","1573-2665"],"issn-type":[{"value":"0141-8955","type":"print"},{"value":"1573-2665","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,5,2]]},"assertion":[{"value":"2022-06-28","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-04-17","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-05-02","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}