{"id":9939,"date":"2023-05-02T12:31:28","date_gmt":"2023-05-02T16:31:28","guid":{"rendered":"https:\/\/mrm.research.mcgill.ca\/2023\/05\/mrm-insights-one-and-done-gene-therapeutics-changing-one-base-at-a-time\/"},"modified":"2023-05-02T15:16:40","modified_gmt":"2023-05-02T19:16:40","slug":"mrm-insights-therapies-geniques-uniques-changer-une-base-a-la-fois","status":"publish","type":"post","link":"https:\/\/mrm.research.mcgill.ca\/fr\/2023\/05\/mrm-insights-therapies-geniques-uniques-changer-une-base-a-la-fois\/","title":{"rendered":"MRM Insights : Th\u00e9rapies g\u00e9niques uniques – changer une base \u00e0 la fois"},"content":{"rendered":"
\"\"

Dre Raquel Cuella Martin<\/p><\/div>\n

Chaque mois, dans les\u00a0MRM Insights<\/a><\/strong>, un membre du R\u00e9seau MRM \u00e9crit sur les cellules souches et la m\u00e9decine r\u00e9g\u00e9n\u00e9rative d\u2019un point de vue diff\u00e9rent. Ce mois-ci, la Dre Raquel Cuella Martin<\/strong>, Professeure adjointe en g\u00e9nomique fonctionnelle au Centre de g\u00e9nomique de McGill, nous parle des th\u00e9rapies g\u00e9niques uniques<\/strong>.<\/p>\n

Th\u00e9rapies g\u00e9niques uniques : changer une base \u00e0 la fois<\/h2>\n

En 2013, la technologie CRISPR \/ Cas9 a \u00e9t\u00e9 utilis\u00e9e pour la premi\u00e8re fois pour modifier le g\u00e9nome humain. Plus de dix ans plus tard, je suis toujours \u00e9merveill\u00e9e de voir o\u00f9 cette technologie peut nous mener. Aujourd\u2019hui, la r\u00e9\u00e9criture de base prend le devant de la sc\u00e8ne, aid\u00e9e par un l\u00e9ger parti pris de ma part.<\/p>\n

Les \u00e9diteurs de base sont le r\u00e9sultat d\u2019une ing\u00e9nierie \u00e9l\u00e9gante des prot\u00e9ines. Fusionnez une nickase Cas9 avec de la cytidine et de l\u2019ad\u00e9nosine d\u00e9saminases et obtenez des substitutions de bases C\u02c3T, A\u02c3G et C\u02c3G \u00e0 l\u2019emplacement g\u00e9nomique o\u00f9 l\u2019ARN guide unique (ARNg) le prendra (1-5). Le c\u00f4t\u00e9 positif, c\u2019est que les \u00e9diteurs de base ins\u00e9reront des mutations avec une grande efficacit\u00e9, m\u00eame dans les cellules diplo\u00efdes. Et plus de cassures d\u2019ADN toxiques caus\u00e9es par Cas9. En revanche, les mutations hors cible sont toujours pr\u00e9sentes, \u00e0 la fois dans l\u2019ADN et l\u2019ARN.<\/p>\n

Si la technologie CRISPR\/Cas9 a d\u00e9j\u00e0 cr\u00e9\u00e9 des remous dans le domaine de la th\u00e9rapie g\u00e9nique, les \u00e9diteurs de base vont plus loin. Est-il possible de simplement corriger les mutations pathog\u00e8nes chez l\u2019homme ? Peut-on concevoir une mutation ponctuelle et inverser durablement les ph\u00e9notypes de la maladie chez les patients atteints de troubles g\u00e9n\u00e9tiques ?<\/p>\n

En 2021, nous avons assist\u00e9 \u00e0 un succ\u00e8s pr\u00e9clinique dans l\u2019utilisation du criblage d\u2019\u00e9dition de base pour le traitement de l\u2019hypercholest\u00e9rol\u00e9mie familiale (6,7). Les auteurs ont cibl\u00e9 PCSK9<\/em>, un g\u00e8ne qui r\u00e9gule le renouvellement du r\u00e9cepteur du cholest\u00e9rol \u00e0 lipoprot\u00e9ines de basse densit\u00e9 (LDL) et, par cons\u00e9quent, l\u2019absorption du cholest\u00e9rol LDL. La raison : des \u00e9tudes g\u00e9n\u00e9tiques avaient r\u00e9v\u00e9l\u00e9 que l\u2019invalidation du g\u00e8ne PCSK9<\/em> chez l\u2019humain causait des niveaux quasi n\u00e9gligeables de cholest\u00e9rol LDL dans le sang et une protection contre les maladies cardiovasculaires (8). L\u2019injection intraveineuse de nanoparticules lipidiques portant un \u00e9diteur de base d\u2019ad\u00e9nosine et un ARNg ciblant le site d\u2019\u00e9pissage de l\u2019exon 1 de PCSK9<\/em> a entra\u00een\u00e9 une efficacit\u00e9 d\u2019\u00e9dition de 70% dans le foie de souris (6,7). Chez les singes cynomolgus, une efficacit\u00e9 d\u2019\u00e9dition de 63% a \u00e9t\u00e9 observ\u00e9e dans le foie, avec des r\u00e9ductions associ\u00e9es de 81 et 65% des taux sanguins de PCSK9 et cholest\u00e9rol LDL respectivement (6). Dans l\u2019\u00e9tude initiale, les singes ont \u00e9t\u00e9 suivis pendant 8 mois, avec une r\u00e9duction stable de PCSK9 et du cholest\u00e9rol LDL (6). Une surveillance plus longue a permis de signaler des r\u00e9ductions stables au jour 476 sans transmission germinale de l\u2019\u00e9dition somatique (9).<\/p>\n

Il y a quelques semaines \u00e0 peine, l\u2019\u00e9dition de base a \u00e9t\u00e9 appliqu\u00e9e \u00e0 une autre maladie g\u00e9n\u00e9tique, l\u2019amyotrophie spinale (AMS). L\u2019AMS est la principale cause de mortalit\u00e9 infantile, caus\u00e9e par la perte homozygote du g\u00e8ne SMN1<\/em>. En l\u2019absence de SMN1, son homologue presque identique SMN2 peut partiellement compenser la perte. Cette \u00ab\u00a0quasi-identit\u00e9\u00a0\u00bb est caus\u00e9e par une mutation sur le site d\u2019\u00e9pissage de l\u2019exon 6 de SMN2<\/em> entra\u00eenant une perte de l\u2019exon 7 et une stabilit\u00e9 r\u00e9duite des prot\u00e9ines. Les traitements de l\u2019AMS sont disponibles selon deux principes : i) le remplacement g\u00e9nique ou ii) favoriser l\u2019inclusion de l\u2019exon7 sur le g\u00e8ne SNM2<\/em>. Bien qu\u2019efficaces, ces traitements (incroyablement !) co\u00fbteux n\u00e9cessitent une administration r\u00e9p\u00e9t\u00e9e tout au long de la vie du patient.<\/p>\n

Un traitement en une fois est-il possible pour les patients atteints d\u2019AMS ? Les laboratoires Kleinstiver et Liu ont offert quelques id\u00e9es ici (10,11).<\/p>\n

Les deux groupes se sont concentr\u00e9s sur la \u00ab\u00a0correction\u00a0\u00bb du g\u00e8ne SNM2<\/em> comme strat\u00e9gie pour restaurer un transcrit SMN comp\u00e9tent, quelle que soit la mutation SMN1<\/em> port\u00e9e par le patient. L\u2019\u00e9diteur de base d\u2019ad\u00e9nosine et l\u2019ARNg ciblant la mutation du site d\u2019\u00e9pissage SNM2<\/em> ont \u00e9t\u00e9 int\u00e9gr\u00e9s dans un syst\u00e8me d\u2019ad\u00e9novirus (AAV9) qui infecte sp\u00e9cifiquement les neurones. L\u2019injection intra-cerebroventriculaire du traitement de r\u00e9\u00e9criture de base dans un mod\u00e8le murin SMA a entra\u00een\u00e9 une correction efficace des mutations pour jusqu\u2019\u00e0 37% des all\u00e8les SNM2<\/em> dans les motoneurones spinaux (11). Les ph\u00e9notypes de la maladie dans les motoneurones spinaux ont \u00e9galement \u00e9t\u00e9 att\u00e9nu\u00e9s et la dur\u00e9e de vie prolong\u00e9e de ~33%. Des r\u00e9sultats encourageants malgr\u00e9 une mise en garde majeure : le m\u00e9canisme intrins\u00e8quement lent de la r\u00e9\u00e9criture de base entrait en conflit avec la fen\u00eatre th\u00e9rapeutique r\u00e9duite pour le traitement de l\u2019AMS chez la souris. Pour contourner cela, le laboratoire Liu a propos\u00e9 la combinaison du traitement de r\u00e9\u00e9criture de base avec la th\u00e9rapie antisens nomm\u00e9e nusinersen, approuv\u00e9e par la FDA. L\u2019id\u00e9e \u00e9tant que l\u2019action rapide du nusinersen augmentera rapidement les niveaux de SMN et \u00e9tendra la fen\u00eatre pour que l\u2019\u00e9diteur de base puisse ins\u00e9rer la modification permanente. Ils ont tir\u00e9 le gros lot ! Les r\u00e9sultats ont \u00e9t\u00e9 consid\u00e9rablement am\u00e9lior\u00e9s. La dur\u00e9e de vie des souris \u00e9dit\u00e9es a \u00e9t\u00e9 multipli\u00e9e par 10, et elles \u00e9taient \u00e9quivalentes \u00e0 celles des souris saines pour 33 param\u00e8tres de la fonction motrice (11).<\/p>\n

Ces r\u00e9sultats nous rapprochent de la promesse de traitements pr\u00e9cis et ponctuels pour les troubles g\u00e9n\u00e9tiques. Des essais cliniques de phase I d\u2019un traitement de r\u00e9\u00e9criture de base pour l\u2019hypercholest\u00e9rol\u00e9mie familiale recrutent d\u2019ailleurs actuellement… Je suppose que nous devrons juste attendre de voir les r\u00e9sultats !<\/p>\n

 <\/p>\n

R\u00e9f\u00e9rences<\/em><\/h3>\n

1. Komor AC, Kim YB, Packer MS, Zuris JA, and Liu DR (2016). Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature 533, 420-424.
\n2. Gaudelli NM, Komor AC, Rees HA, Packer MS, Badran AH, Bryson DI, et al. (2017). Programmable base editing of A\u2022T to G\u2022C in genomic DNA without DNA cleavage. Nature 551, 464-471.
\n3. Zhao D, Li J, Li S, Xin X, Hu M, Price MA, et al. (2021). Glycosylase base editors enable C-to-A and C-to-G base changes. Nat Biotechnol 39, 35-40.
\n4. Kurt IC, Zhou R, Iyer S, Garcia SP, Miller BR, Langner LM, et al. (2021). CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells. Nat Biotechnol 39, 41-46.
\n5. Koblan LW, Arbab M, Shen MW, Hussmann JA, Anzalone AV, Doman, JL et al. (2021). Efficient C\u2022G-to-G\u2022C base editors developed using CRISPRi screens, target-library analysis, and machine learning. Nat Biotechnol 39, 1414-1425.
\n6. Musunuru K, Chadwick AC, Mizoguchi T, Garcia SP, DeNizio JE, Reiss CW, et al. (2021). In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates. Nature 593, 429-434.
\n7. Rothgangl T, Dennis MK, Lin PJC, Oka R, Witzigmann D, Villiger L, et al. (2021). In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels. Nat Biotechnol 39, 949-957.
\n8. Cohen JC, Boerwinkle E, Mosley TH Jr, and Hobbs HH (2006). Sequence variations in PCSK9, low LDL, and protection against coronary heart disease. N Engl J Med 354, 1264-1272.
\n9. Lee RG, Mazzola AM, Braun MC, Platt C, Vafai SB, Kathiresan S, et al. (2023). Efficacy and Safety of an Investigational Single-Course CRISPR Base-Editing Therapy Targeting PCSK9 in Nonhuman Primate and Mouse Models. Circulation 147, 242-253.
\n10. Alves CRR, Ha LL, Yaworski R, Lazzarotto CR, Christie KA, Reilly A, et al. (2023). Base editing as a genetic treatment for spinal muscular atrophy. BioRxiv, 524978.
\n11. Arbab M, Matuszek Z, Kray KM, Du A, Newby GA, Blatnik AJ, et al. (2023). Base editing rescue of spinal muscular atrophy in cells and in mice. Science eadg6518. Epub ahead of print.<\/p>\n","protected":false},"excerpt":{"rendered":"

Chaque mois, dans les\u00a0MRM Insights, un membre du R\u00e9seau MRM \u00e9crit sur les cellules souches…<\/p>\n","protected":false},"author":6,"featured_media":9946,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_bbp_topic_count":0,"_bbp_reply_count":0,"_bbp_total_topic_count":0,"_bbp_total_reply_count":0,"_bbp_voice_count":0,"_bbp_anonymous_reply_count":0,"_bbp_topic_count_hidden":0,"_bbp_reply_count_hidden":0,"_bbp_forum_subforum_count":0,"ngg_post_thumbnail":0,"footnotes":""},"categories":[162],"tags":[689,164,178,446],"class_list":["post-9939","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-mrm-insights-fr","tag-gene-therapeutics-fr","tag-mrm-fr","tag-mrm-insights-fr","tag-therapie-genique"],"acf":[],"yoast_head":"\nMRM Insights : Th\u00e9rapies g\u00e9niques uniques - changer une base \u00e0 la fois | MRM<\/title>\n<meta name=\"description\" content=\"Chaque 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