LENTIVIRAL-MEDIATED GENE THERAPY FOR SEVERE PYRUVATE KINASE DEFICIENCY: RESULTS FROM AN ONGOING GLOBAL PHASE 1 STUDY

被引：0

作者：

Lopez Lorenzo, Jose Luis ^{[1
,2
]}

Shah, Ami ^{[3
]}

Sevilla, Julian ^{[5
,6
]}

Navarro, Susana ^{[2
,6
,7
]}

Llanos, Lucia ^{[1
,2
]}

de Camino Gaisse, Begona Perez ^{[1
,2
]}

Sanchez, Sol ^{[1
,2
]}

Zubicaray, Josune ^{[5
]}

Glader, Bertil ^{[3
,4
]}

Chien, May ^{[3
,4
]}

Quintana Bustamante, Oscar ^{[2
,6
,7
]}

Zeini, Miriam ^{[8
]}

Choi, Grace ^{[8
]}

Nicoletti, Eileen ^{[8
]}

Rao, Gayatri ^{[8
]}

Roncarolo, Maria Grazia ^{[3
,4
]}

Bueren, Juan ^{[2
,6
,7
]}

Schwartz, Jonathan ^{[8
]}

Carlos Segovia, Jose ^{[2
,6
,7
]}

机构：

[1] Hosp Univ Fdn Jimenez Diaz, Madrid, Spain

[2] Inst Invest Sanitaria Fdn Jimenez Diaz IIS FJD, Madrid, Spain

[3] Stanford Univ, Sch Med, Stanford, CA USA

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

[5] Hosp Infantil Univ Nino Jesus HIUNJ, Fdn invest Biomed, Madrid, Spain

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

[7] Ctr Invest Energet Medioambientales & Tecnol CIEM, Madrid, Spain

[8] Rocket Pharmaceut Inc, Cranbury, NJ USA

来源：

BONE MARROW TRANSPLANTATION | 2023年 / 58卷 / SUPP1期

关键词：

D O I：

暂无

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

P181

引用

页码：275 / 276

页数：2

共 50 条

[1] Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study
Shah, Ami J.
Lopez Lorenzo, Jose Luis
Sevilla, Julian
Navarro, Susana
Llanos, Lucia
de Camino Gaisse, Begona Perez
Sanchez, Sol
Zubicaray, Josune
Glader, Bert
Chien, May
Bustamante, Oscar Quintana
Zeini, Miriam
Choi, Grace
Nicoletti, Eileen
Rao, Gayatri R.
Grazia Roncarolo, Maria
Bueren, Juan A.
Schwartz, Jonathan D.
Segovia, Jose C.
BLOOD, 2022, 140 : 4902 - 4903
[2] LENTIVIRAL-MEDIATED GENE THERAPY FOR SEVERE PYRUVATE KINASE DEFICIENCY: GLOBAL PHASE 1 STUDY RESULTS
Shah, Ami
Lorenzo, Jose Luis Lopez
Sevilla, J.
Navarro, Susana
Llanos, Lucia
Gaisse, Begona Perez De Camino
Sanchez, Sol
Zubicaray, Josune
Glader, Bertil
Chien, May
Bustamante, OQuintana
Zeini, Miriam
Choi, Grace
Nicoletti, Eileen
Rao, Gayatri
Roncarolo, Maria Grazia
Bueren, Juan
Schwartz, J.
Segovia, Jose Carlos
PEDIATRIC BLOOD & CANCER, 2023, 70 : S133 - S134
[3] Interim Results from an Ongoing Global Phase 1 Study of Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency
Lopez Lorenzo, J. L.
Shah, A. J.
Sevilla, J.
Navarro, S.
Llanos, L.
de Camino Gaisse, B. Perez
Sanchez, S.
Zubicaray, J.
Glader, B.
Chien, M.
Bustamante, O.
Zeini, M.
Choi, G.
Nicoletti, E.
Rao, G. R.
Roncarolo, M. G.
Bueren, J. A.
Schwartz, J. D.
Segovia, J. C.
HUMAN GENE THERAPY, 2022, 33 (23-24) : A58 - A58
[4] Global phase 1 study results of lentiviral mediated gene therapy for severe pyruvate kinase deficiency
Sevilla, J.
Shah, A. J.
Lorenzo, J. L. L.
Navarro, S.
Lanos, L.
de Camino Gaisse, B. P.
Sanchez, S.
Zubicaray, J.
Glader, B.
Chien, M.
Bustamante, O. Q.
Zeini, M.
Choi, G.
Nicoletti, E.
Rao, G. R.
Roncarolo, M. G.
Bueren, J. A.
Schwartz, J. D.
Segovia, J. C.
HUMAN GENE THERAPY, 2024, 35 (3-4) : A4 - A4
[5] Global Phase 1 Study Results of Lentiviral Mediated Gene Therapy for Severe Pyruvate Kinase Deficiency
Shah, Ami J.
Lopez Lorenzo, Jose Luis
Sevilla, Julian
Navarro, Susana
Llanos, Lucia
de Camino Gaisse, Begona Perez
Sanchez, Sol
Zubicaray, Josune
Glader, Bertil
Chien, May
Quintana Bustamante, Oscar
Zeini, Miriam
Choi, Grace
Nicoletti, Eileen
Rao, Gayatri
Roncarolo, Maria-Grazia
Bueren, Juan A.
Schwartz, Jonathan
Carlos Segovia, Jose
MOLECULAR THERAPY, 2023, 31 (04) : 118 - 119
[6] Changing the Treatment Paradigm for Pyruvate Kinase Deficiency with Lentiviral Mediated Gene Therapy: Interim Results from an Ongoing Global Phase 1 Study
Shah, A. J.
Lopez Lorenzo, J. L.
Navarro, S.
Sevilla, J.
Llanos, L.
de Camino Gaisse, B. Perez
Sanchez, S.
Glader, B.
Chien, M.
Quintana Bustamante, O.
Zeini, M.
Choi, G.
Nicoletti, E.
Rao, G. R.
Roncarolo, M. G.
Bueren, J. A.
Schwartz, J. D.
Segovia, J. C.
MOLECULAR THERAPY, 2022, 30 (04) : 170 - 170
[7] Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients
Shah, Ami J.
Lopez Lorenzo, Jose Luis
Navarro, Susana
Sevilla, Julian
Llanos, Lucia
Perez de Camino Gaisse, Begona
Sanchez, Sol
Glader, Bert
Chien, May
Quintana Bustamante, Oscar
Beard, Brian C.
Law, Kenneth M.
Zeini, Miriam
Choi, Grace
Nicoletti, Eileen
Rao, Gayatri R.
Grazia Roncarolo, Maria
Bueren, Juan A.
Schwartz, Jonathan D.
Segovia, Jose C.
BLOOD, 2021, 138
[8] Lentiviral mediated gene therapy for pyruvate kinase deficiency: Interim results of a global phase 1 study for adult and pediatric patients
Lorenzo, J. L. Lopez
Shah, A. J.
Navarro, S.
Sevilla, J.
Llanos, L.
Gaisse, B. P. de Camino
Sanchez, S.
Glader, B.
Chien, M.
Bustamante, O. Quintana
Beard, B. C.
Zeini, M.
Choi, G.
Nicoletti, E.
Rao, G. R.
Roncarolo, M. G.
Bueren, J. A.
Schwartz, J.
Segovia, J. C.
HUMAN GENE THERAPY, 2021, 32 (19-20) : A25 - A25
[9] Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric Patients
Lopez Lorenzo, Jose Luis
Shah, Ami J.
Navarro, Susana
Sevilla, Julian
Llanos, Lucia
Perez Camino de Gaisse, Begona
Sanchez, Sol
Glader, Bertil
Chien, May
Quintana-Bustamante, Oscar
Beard, Brian C.
Law, Kenneth M.
Zeini, Miriam
Choi, Grace
Nicoletti, Eileen
Rao, Gayatri R.
Roncarolo, Maria Grazia
Bueren, Juan A.
Schwartz, Jonathan D.
Segovia, Jose C.
MOLECULAR THERAPY, 2021, 29 (04) : 42 - 43
[10] Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients
Lorenzo, Jose Luis Lopez
Navarro, Susana
Shah, Ami J.
Roncarolo, Maria Grazia
Sevilla, Julian
Llanos, Lucia
de Gaisse, Begona Perez Camino
Sanchez, Sol
Glader, Bertil
Chien, May
Bustamante, Oscar Quintana
Beard, Brian C.
Law, Kenneth M.
Zeini, Miriam
Choi, Grace
Nicoletti, Eileen
Bueren, Juan A.
Rao, Gayatri R.
Schwartz, Jonathan D.
Segovia, Jose C.
BLOOD, 2020, 136

← 1 2 3 4 5 →