Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RP-L301

被引：0

作者：

Shah, Ami ^{[1
,2
,3
]}

Sevilla, Julian ^{[4
,5
]}

Lopez Lorenzo, Jose Luis ^{[6
,7
]}

Navarro, Susana ^{[5
,7
,8
]}

Llanos, Lucia ^{[6
,7
]}

de Camino Gaisse, Belgona Perez ^{[6
,7
]}

Sanchez, Sol ^{[6
,7
]}

Zubicaray, Josune ^{[4
]}

Glader, Bert ^{[2
,3
]}

Chien, May ^{[2
,3
]}

Quintana Bustamante, Oscar ^{[5
,7
,8
]}

Gonzalez Vicent, Marta ^{[4
,5
]}

Iriondo, June ^{[4
,5
]}

Rothe, Michael ^{[9
]}

John-Neek, Philipp ^{[9
]}

Bastone, Antonella ^{[9
]}

Schambach, Axel ^{[9
]}

Zeini, Miriam ^{[10
]}

Choi, Grace ^{[10
]}

Nicoletti, Eileen ^{[10
]}

Rao, Gayatri ^{[10
]}

Roncarolo, Maria-Grazia ^{[1
,2
,3
]}

Bueren, Juan A. ^{[5
,7
,8
]}

Schwartz, Jonathan ^{[10
]}

Segovia, Jose-Carlos ^{[5
,7
,8
]}

机构：

[1] Stanford Univ, Ctr Definit & Curat Med, Stanford, CA USA

[2] Stanford Univ, Sch Med, Div Hematol Oncol Stem Cell Transplant Regenerat, Dept Pediat, Stanford, CA USA

[3] Lucile Packard Childrens Hosp, Palo Alto, CA USA

[4] HIUNJ, Fdn Invest Biomed, Hematol & Hemoterapia, Madrid, Spain

[5] Ctr Invest Biomed Red Enfermedades Raras CIBERER, Madrid, Spain

[6] Hosp Univ Fdn Jimenez Diaz, Inst Invest Sanitaria Fdn Jimenez Diaz IIS FJD, Madrid, Spain

[7] Inst Invest Sanitaria Fdn Jimenez Diaz IIS FJD, Unidad Mixta Terapias Avanzadas, Madrid, Spain

[8] Ctr Invest Energet Medioambientales & Tecnol CIEM, Unidad Innovac Biomed, Madrid, Spain

[9] Hannover Med Sch, Hannover, Germany

[10] Rocket Pharmaceut, Cranbury, NJ USA

来源：

MOLECULAR THERAPY | 2024年 / 32卷 / 04期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

引用

页码：2 / 3

页数：2

共 50 条

[41] LENTIVIRAL-MEDIATED GENE THERAPY FOR FANCONI ANEMIA [GROUP A]: RESULTS FROM RP-L102 CLINICAL TRIALS
Czechowicz, Agnieszka
Sevilla, Julian
Booth, Claire
Agarwal, Rajni
Zubicaray, Josune
Rio, Paula
Navarro, Susana
Chetty, Kritika
O'Toole, Grainne
Xu-Bayford, Jinhua
Ancliff, Philip
Sebastian, Elena
Choi, Grace
Zeini, Miriam
Nicoletti, Eileen
Wagner, John
Eide, Cindy
Rao, Gayatri
Thrasher, Adrian
Schwartz, Jonathan
Roncarolo, Maria Grazia
Bueren, Juan
PEDIATRIC BLOOD & CANCER, 2023, 70 : S136 - S137
[42] ERYTHROCYTE PYRUVATE-KINASE DEFICIENCY - A KINETIC-STUDY OF THE MEMBRANE-LOCALIZED AND CYTOPLASMATIC ENZYME FROM 6 PATIENTS
LAKOMEK, M
TILLMANN, W
SCHARNETZKY, M
SCHROTER, W
WINKLER, H
ENZYME, 1983, 29 (03) : 189 - 197
[43] GLOBAL PREVALENCE OF PEDIATRIC AND ADULT IGE-MEDIATED FOOD ALLERGIES: RESULTS: FROM THE ASSESS FA STUDY
Gupta, R.
Marvel, J.
Tassinari, P.
Mnif, T.
Hleyhel, M.
Vincent, B.
Worm, M.
Fiocchi, A.
ANNALS OF ALLERGY ASTHMA & IMMUNOLOGY, 2023, 131 (05) : S7 - S8
[44] A Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Initial Results from the First Treated Patient
Kohn, Donald
Rao, Gayatri
Almarza, Elena
Terrazas, Dayna
Nicoletti, Eileen
Fernandes, Augustine
Kuo, Caroline
De Oliveira, Satiro
Moore, Theodore
Law, Ken
Beard, Brian
Sevilla, Julian
Mesa, Christina
Bueren, Juan
Schwartz, Jonathan
JOURNAL OF CLINICAL IMMUNOLOGY, 2020, 40 (SUPPL 1) : S103 - S103
[45] A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Initial Results from the First Treated Patient
Kohn, Donald
Rao, Gayatri
Almarza, Elena
Terrazas, Dayna
Nicoletti, Eileen
Fernandes, Augustine
Kuo, Caroline
De Oliveira, Satiro
Moore, Theodore
Law, Ken
Beard, Brian
Sevilla, Julian
Mesa-Nunez, Cristina
Bueren, Juan
Schwartz, Jonathan
MOLECULAR THERAPY, 2020, 28 (04) : 56 - 57
[46] Generation of transgene-free hiPSCs from peripheral blood of Pyruvate Kinase Deficiency patients for gene correction by Meganuclease mediated Homologous Recombination
Garate, Zita
Quintana-Bustamante, Oscar
Cerrato, Laura
Galetto, Roman
Rodriguez-Fornes, Fatima
Poirot, Luren
Maia, Tabita
Ribeiro, Leticia
Fusaki, Noemi
Guenechea, Guillermo
Gouble, Agnes
Bueren, Juan
Carlos Segovia, Jose
HUMAN GENE THERAPY, 2012, 23 (10) : A87 - A87
[47] Outcome Following Infliximab Therapy in Pediatric Patients with Ulcerative Colitis: Results from a Multicenter Study
Hyams, Jeffrey S.
Lerer, Trudy
Griffiths, Anne M.
Evans, Jonathan S.
Carvalho, Ryan S.
Otley, Anthony R.
Bousvaros, Athos
Mack, David R.
Pfefferkorn, Marian D.
Rosh, Joel R.
Kay, Marsha H.
Crandall, Wallace
Tomer, Gitit
Leleiko, Neal S.
Keljo, David J.
Markowitz, James
GASTROENTEROLOGY, 2009, 136 (05) : A673 - A673
[48] Practice of volume therapy in patients with severe sepsis: results from a nationwide sepsis prevalence study
Hartog, Christiane S.
Brunkhorst, Frank M.
Bloos, Frank
Bogatsch, Holger
Engel, Christoph
Sengebusch, Kerstin
Reinhart, Konrad
Ragaller, Maximilian
INTENSIVE CARE MEDICINE, 2010, 36 (03) : 553 - 554
[49] Practice of volume therapy in patients with severe sepsis: results from a nationwide sepsis prevalence study
Christiane S. Hartog
Frank M. Brunkhorst
Frank Bloos
Holger Bogatsch
Christoph Engel
Kerstin Sengebusch
Konrad Reinhart
Maximilian Ragaller
Intensive Care Medicine, 2010, 36 : 553 - 554
[50] EFFICACY AND SAFETY OF MIRIKIZUMAB THERAPY IN PEDIATRIC PATIENTS WITH MODERATE TO SEVERE ULCERATIVE COLITIS: FINAL RESULTS FROM THE PHASE 2 SHINE-1 STUDY
Kaplan, Jess
Bousvaros, Athos
Turner, Dan
Dubinsky, Marla C.
Komocsar, Wendy
Larkin, Amy E.
Johns, Jordan
Zhang, Xin
Crandall, Wallace
Arora, Vipin
Todd, Kris
Hyams, Jeffrey S.
GASTROENTEROLOGY, 2024, 166 (05) : S819 - S820

← 1 2 3 4 5 →