Resous rekòmande yo

Yon konpilasyon nan Terapi jèn nan papye syantifik nan emofil, chwazi esè klinik, dokiman regilasyon ak lyen itil

Papye syantifik ak dokiman regilasyon

Revizyon Jenoterapi pou Hemofili

1. Abbasi J. Terapi jèn pou emofil montre pwomès. JAMA. 2018; 319 (6): 539. https://www.ncbi.nlm.nih.gov/pubmed/29450507

2. Arruda VR, et al. Nouvo apwòch pou terapi emofili: siksè ak defi. San. 2017; 130: 2251-2256. https://www.ncbi.nlm.nih.gov/pubmed/29018078

3. balansansh P, et al. Nouvo terapi ak aktyèl pwogrè klinik nan hemofili A. Ther Adv Ematol.2018; 9: 49-61. https://www.ncbi.nlm.nih.gov/pubmed/?term=29387330

4. Beck DL. Avans nan terapi jèn pou emofili. Ash Klinik Nouvèl. Oktòb NAN, NAN. https://www.ashclinicalnews.org/features/breakthroughs-gene-therapy-hemophilia/

5. Colella P, et al. Emerging pwoblèm nan AAV-medyatè nan terapi jèn vivo. Mol Ther Meth Clin Dev. 2018; 8: 87-104. https://www.ncbi.nlm.nih.gov/pubmed/29326962

6. DiMichele DM. Navige vit monte desann sou gran wout inovasyon nan terapi emofil. HemaSphere. KOULYE: KOUMAN POU: SEKSYON. https://www.ncbi.nlm.nih.gov/pubmed/30887008

7. Doshi BS, et al. Terapi jèn pou emofili: kisa lavni an kenbe? Ther Adv Ematol. 2018; 9: 273-293. https://www.ncbi.nlm.nih.gov/pubmed/30210756

8. Dunbar CE, et al. Terapi jèn vini nan laj. Syans. 2018; 359: 4672. https://www.ncbi.nlm.nih.gov/pubmed/29326244

9. Evens H, et al. Terapi jèn emofilik: Soti nan pyèj anbale pou gamechanger. Emofili. 2018;24(Suppl. 6):50–59. https://www.ncbi.nlm.nih.gov/pubmed/29878653

10. George LA. Terapi jèn nan emofil vini ki gen laj. Pwogram Edikasyon Hematoloji Am Soc Hematol. 2017; 587-594. https://www.ncbi.nlm.nih.gov/pubmed/29222308

11. Mahlangu J, et al. Terapi kap fè emosyonèl pou pèspektiv global. Emofili. 2018;24(Suppl. 6):15–21. https://www.ncbi.nlm.nih.gov/pubmed/29878661

12. Makris M. Yon laj an lò pou tretman emofili? Emofili. 2018; 24: 175-176. https://www.ncbi.nlm.nih.gov/pubmed/29601683

13. Park CY, et al. Genomic-koreksyon teknoloji pou jèn koreksyon nan emofili. Imèn Jen. 2016; 135: 977-981. https://www.ncbi.nlm.nih.gov/pubmed/27357631

14. Perrin GQ, et al. Mizajou sou terapi jèn pou emofili. San. 2019; 133: 407-414. https://www.ncbi.nlm.nih.gov/pubmed/30559260

15. Pierce GF, et al. Yon multitude nan terapi anba etid pou emofili. Mol Ther. 2017; 25: 2429-2430. https://www.ncbi.nlm.nih.gov/pubmed/29055619

16. Pierce GF, et al. Pase, prezan ak tan kap vini nan terapi jèn emofilik: Soti nan vektè ak transjèn rive rezilta li te ye ak enkoni. Emofili. 2018;24(Suppl. 6):60–67. https://www.ncbi.nlm.nih.gov/pubmed/29878660

17. Pierce GF, et al. Premye WFH jèn terapi tab wonn tab la: Konprann peyizaj la ak defi nan terapi jèn pou emofili atravè mond lan. Emofili. 2019; 1–6. https://www.ncbi.nlm.nih.gov/pubmed/30604914

18. Tiyo SW. Nouvo terapi pou emofili. Pwogram Edikasyon Hematoloji Am Soc Hematol. 2016; (1): 650-656. https://www.ncbi.nlm.nih.gov/pubmed/27913542

19. Tiyo SW. Terapi jèn pou emofili. Pedyat Kansè nan san. KOULYE: KOUMAN POU: SEKSYON. https://www.ncbi.nlm.nih.gov/pubmed/29077262

20. Schutgens R, et al. Terapi jèn nan emofili: Soti nan battage a nan espwa. HemaSphere. 2018;2(2):22:e37. https://journals.lww.com/hemasphere/Fulltext/2018/04000

Lyen entent itil

Fondasyon Nasyonal pou Hemofili
https://www.hemophilia.org/

Mondyal Federasyon nan Emofili
https://www.wfh.org/en/page.aspx?pid=492

Pwogrè nan klinik nan Terapi Gene pou Emofili
https://www.medscape.org/sites/advances/gene-therapy-hemophilia

Seleksyone esè klinik yo

Hemofilya A

  • NCT02576795. Yon Faz 1/2, Dòz-Eskalasyon, Sekirite Sosyal, Tolerans ak Efikasite Etid sou Rokarovovec Valoctocogene, yon viris ki asosye ak Adenovirus-vektè ki gen medyatè Transfè jeni nan VIII Imèn Faktè nan pasyan ki gen imès grav A. https://clinicaltrials.gov/ct2/show/NCT02576795
  • NCT03520712. Yon Faz 1/2 Sekirite Sosyal, Tolerans, ak efikasite etid sou Rokarovovec Valoctocogene, yon viris ki asosye-Associated vektè-médiésye Gene Transfere nan VIII Imèn Faktè nan pasyan Hemophilia A ki gen rezidyèl FVIII Nivo ≤ 1 IU / dL ak antikò pre-egziste ki kont AAV5 . https://clinicaltrials.gov/ct2/show/NCT03520712
  • NCT03392974. Faz 3 Etid Evalye Efficacité / Sekirite nan Valoctocogene Roxaparvovec yon AAV vektè-medyatè Gene Transfè nan hFVIII nan yon Dòz nan 4E13vg / kg nan Hemophilia A Pasyan ki gen rezidyèl FVIII Nivo ≤1IU / dL ki resevwa profiletik FVIII perfusion. https://clinicaltrials.gov/ct2/show/NCT03392974
  • NCT03370913. Yon Faz 3 Open-Label, Single-bra etidye Evalye efikasite a ak Sekirite nan BMN 270, yon viris ki asosye-Associated vektè ki gen medyatè transfè jeni nan VIII Imèn Faktè nan pasyan Hemophilia A ki gen rezidyèl FVIII Nivo ≤ 1 IU / dL ki resevwa pwofilaktik Perfusion FVIII. https://clinicaltrials.gov/ct2/show/NCT03370913
  • NCT03432520. Yon Evalyasyon milti-sant nan alontèm Sekirite Sosyal la ak efikasite nan SPK-8011 [Adeno-Associated vektè viral Avèk B-Domèn Efase moun jèn VIII Faktè] nan Gason ki gen emofil A. https://clinicaltrials.gov/ct2/show/NCT03432520
  • NCT03003533. Transfè jèn, Dòz-Jwenn Sekirite, Tolerans, ak efikasite Etid la SPK-8011 [yon recombinant Adeno-Associated vektè viral Avèk Imen faktè Imen VIII] nan moun ki gen emofil A. https://clinicaltrials.gov/ct2/show/NCT03003533
  • NCT03734588. Etid Dòz-pou jwenn nan Terapi jèm SPK-8016 nan pasyan ki gen emofil A pou sipòte evalyasyon nan moun ki gen inhibiteur FVIII. https://clinicaltrials.gov/ct2/show/NCT03734588
  • NCT03370172. Yon Global, Open-Label, milti-sant, Faz 1/2 etid sou Sekirite Sosyal la ak Dòz la nan BAX 888, yon Adeno-Associated Serotip viris 8 (AAV8) vektè eksprime B-Domèn Efase VIII efase (BDD-FVIII) nan emofil grav. Yon sijè administre yon sèl perfusion vennsèn. https://clinicaltrials.gov/ct2/show/NCT03370172
  • NCT03061201. Yon Faz 1/2, Louvri-etikèt, Adaptatif, etid Dòz-K ap evalye Sekirite Sosyal la ak Tolerabilite nan SB-525 (Recombinant AAV2 / 6 Imèn Faktè Terapi jèn 8 moun) nan granmoun pou Timoun ki gen emofil grav .. https://clinicaltrials.gov/ct2/show/NCT03061201
  • NCT03001830. GO-8: Terapi jèn pou emofil A lè l sèvi avèk yon serotip 8 Kadsid pseudotyped Adeno ki asosye ak vektè Vajen kodaj Faktè VIII-V3. https://clinicaltrials.gov/ct2/show/NCT03001830
  • NCT03217032. Terapi lantiy FVIII Lentiviral pou Hemofili A. https://clinicaltrials.gov/ct2/show/NCT03217032
  • NCT03818763. Faz I etidye Evalye Sekirite ak Posibilite Transfè Gene Twopilatèn ematopyotik ki sible akouchman faktè VIII nan platelèt pou pasyan ki gen emofil A. https://clinicaltrials.gov/ct2/show/NCT03818763

Hemofil B

  • NCT02484092. Terapi jèn, Open-Label, etid Dòz-Eskalasyon nan SPK-9001 [Adeno-Associated vektè viral Avèk Imèn moun faktè IX] Nan sijè ki gen emofil B. https://clinicaltrials.gov/ct2/show/NCT02484092
  • NCT03307980. Ranpli Etid Long: Yon Faktè IX (ranje) Transfè Gene, milti-sant Evalyasyon nan tèm long Sekirite Sosyal ak efikasite nan SPK9001 https://clinicaltrials.gov/ct2/show/NCT03307980
  • NCT02396342. Yon Faz I / II, Open-mete etikèt sou, san kontwòl, Single-dòz, Dòz-moute, Multi-sant Jijman Mennen ankèt sou yon Adeno-ki asosye vektè viral ki gen yon Codon-optimize Imèn moun faktè IX (AAV5-hFIX) administre nan pasyan granmoun ki gen Emofili B. ki Grav oswa ki Modere. https://clinicaltrials.gov/ct2/show/NCT02396342
  • NCT03569891. Faz III, Open-mete etikèt sou, Single-dòz, milti-sant, multinasyonal Jijman Mennen ankèt sou yon serotip 5 vektè Adeno-ki asosye viral ki gen Variant a Padua nan yon Codon-optimize Imèn moun faktè IX-(AAV5-hFIXco-Padova, AMT-061) Administre soufè adilt ki gen emofil B. grav oswa modere grav. https://clinicaltrials.gov/ct2/show/NCT03569891
  • NCT03489291. Faz IIb, Open-mete etikèt sou, Single-dòz, Single-bra, milti-sant Jijman Konfime Faktè IX la Aktivite Nivo nan serotip 5 Adeno ki asosye vektè viral ki gen Variant la Padua nan yon Codon-optimize Imèn Faktè Imèn Codon (AAV5 -hFIXco-Padova, AMT-061) administre soufè adilt ki gen emofil gra oswa modere grav B. https://clinicaltrials.gov/ct2/show/NCT03489291
  • NCT01687608. Yon Faz 1/2 Open-Label, Single Single Dòz Jij la nan yon oto-konplemante optimize Adeno ki asosye viris Serotip 8 Faktè IX Terapi jèn (AskBio009) nan granmoun ki gen emofil B. https://clinicaltrials.gov/ct2/show/NCT01687608
  • NCT03369444. Yon Faz I / II, Louvri etikèt, Multicentre, Dòz sèl ki monte, Etid sou sekirite nan yon Novè Adeno-Associated vektè viral (FLT180a) nan pasyan ki gen emofil B. https://clinicaltrials.gov/ct2/show/NCT03369444
  • NCT03641703. Yon etid Open-etikèt, multikentrik, alontèm pou envestige sou sekirite ak durabilite nan repons apre Dosaj nan yon Novè Adeno ki asosye-vektè viral (FLT180a) nan pasyan ki gen emofil B. https://clinicaltrials.gov/ct2/show/NCT03641703
  • NCT02695160. Yon Etid Faz I, Open-Etikèt, Dòz Moute Pou Evalye Sekirite ak Tolerabilite AAV2 / 6 Faktè IX Terapi Jen Genyen Atravè Zin Nuklea Dwèt (ZFN) Entèmedyè ki se Medyatè Sible SB-FIX nan sijè pou granmoun ki gen emofil grav B. https://clinicaltrials.gov/ct2/show/NCT02695160
  • NCT00979238. Yon etid ouvri sou etikèt dòz Dòz yon vektè Auto-konplemantè ki asosye ak Adeno (scAAV 2/8-LP1-hFIXco) pou transfè jèn nan emofil B. https://clinicaltrials.gov/ct2/show/NCT00979238