共 107 条
[1]
Hematopoietic Cell Transplantation for Mucopolysaccharidosis Patients Is Safe and Effective: Results after Implementation of International Guidelines
[J].
Aldenhoven, Mieke
;
Jones, Simon A.
;
Bonney, Denise
;
Borrill, Roisin E.
;
Coussons, Mary
;
Mercer, Jean
;
Bierings, Marc B.
;
Versluys, Birgitta
;
van Hasselt, Peter M.
;
Wijburg, Frits A.
;
van der Ploeg, Ans T.
;
Wynn, Robert F.
;
Boelens, Jaap Jan
.
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION,
2015, 21 (06)
:1106-1109

Aldenhoven, Mieke
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

Jones, Simon A.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Manchester, Cent Manchester Univ Hosp, Manchester Ctr Genom Med, Willink Unit, Manchester, Lancs, England Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

Bonney, Denise
论文数: 0 引用数: 0
h-index: 0
机构:
Royal Manchester Childrens Hosp, Blood & Marrow Transplant Unit, Manchester M27 1HA, Lancs, England Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

Borrill, Roisin E.
论文数: 0 引用数: 0
h-index: 0
机构:
Royal Manchester Childrens Hosp, Blood & Marrow Transplant Unit, Manchester M27 1HA, Lancs, England Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

Coussons, Mary
论文数: 0 引用数: 0
h-index: 0
机构:
Royal Manchester Childrens Hosp, Blood & Marrow Transplant Unit, Manchester M27 1HA, Lancs, England Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

Mercer, Jean
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Manchester, Cent Manchester Univ Hosp, Manchester Ctr Genom Med, Willink Unit, Manchester, Lancs, England Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

Bierings, Marc B.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

Versluys, Birgitta
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

van Hasselt, Peter M.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Med Ctr Utrecht, Dept Metab Disorders, Utrecht, Netherlands Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

Wijburg, Frits A.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Amsterdam, Dept Pediat, Amsterdam, Netherlands
Univ Amsterdam, Amsterdam Lysosome Ctr Sphinx, Amsterdam, Netherlands Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

van der Ploeg, Ans T.
论文数: 0 引用数: 0
h-index: 0
机构:
Erasmus MC Univ Med Ctr, Ctr Lysosomal & Metab Dis, Rotterdam, Netherlands Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

Wynn, Robert F.
论文数: 0 引用数: 0
h-index: 0
机构:
Royal Manchester Childrens Hosp, Blood & Marrow Transplant Unit, Manchester M27 1HA, Lancs, England Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands

Boelens, Jaap Jan
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands Univ Med Ctr Utrecht, Pediat Blood & Marrow Transplantat Program, NL-3508 AB Utrecht, Netherlands
[2]
Cord blood is the optimal graft source for the treatment of pediatric patients with lysosomal storage diseases: clinical outcomes and future directions
[J].
Aldenhoven, Mieke
;
Kurtzberg, Joanne
.
CYTOTHERAPY,
2015, 17 (06)
:765-774

Aldenhoven, Mieke
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Med Ctr Utrecht, Dept Pediat, Blood & Marrow Transplantat Program, Utrecht, Netherlands Univ Med Ctr Utrecht, Dept Pediat, Blood & Marrow Transplantat Program, Utrecht, Netherlands

Kurtzberg, Joanne
论文数: 0 引用数: 0
h-index: 0
机构:
Duke Univ, Pediat Blood & Marrow Transplant Program, Durham, NC 27710 USA Univ Med Ctr Utrecht, Dept Pediat, Blood & Marrow Transplantat Program, Utrecht, Netherlands
[3]
Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
[J].
Amoasii, Leonela
;
Long, Chengzu
;
Li, Hui
;
Mireault, Alex A.
;
Shelton, John M.
;
Sanchez-Ortiz, Efrain
;
McAnally, John R.
;
Bhattacharyya, Samadrita
;
Schmidt, Florian
;
Grimm, Dirk
;
Hauschka, Stephen D.
;
Bassel-Duby, Rhonda
;
Olson, Eric N.
.
SCIENCE TRANSLATIONAL MEDICINE,
2017, 9 (418)

Amoasii, Leonela
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Long, Chengzu
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA
NYU, Sch Med, Leon H Charney Div Cardiol, New York, NY 10016 USA Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Li, Hui
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Mireault, Alex A.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Shelton, John M.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Southwestern Med Ctr Dallas, Dept Internal Med, Dallas, TX 75390 USA Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Sanchez-Ortiz, Efrain
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

McAnally, John R.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Bhattacharyya, Samadrita
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Schmidt, Florian
论文数: 0 引用数: 0
h-index: 0
机构:
Heidelberg Univ Hosp, BioQuant Ctr, DZIF Partner, Ctr Infect Dis,Virol,Cluster Excellence Cell Netw, D-69120 Heidelberg, Germany Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Grimm, Dirk
论文数: 0 引用数: 0
h-index: 0
机构:
Heidelberg Univ Hosp, BioQuant Ctr, DZIF Partner, Ctr Infect Dis,Virol,Cluster Excellence Cell Netw, D-69120 Heidelberg, Germany Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Hauschka, Stephen D.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Washington, Dept Biochem, Seattle, WA 98195 USA Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Bassel-Duby, Rhonda
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Olson, Eric N.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Dallas, Senator Paul D Wellstone Muscular Dystrophy Coope, Hamon Ctr Regenerat Sci & Med, Southern Med Ctr,Dept Mol Biol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA
[4]
Search-and-replace genome editing without double-strand breaks or donor DNA
[J].
Anzalone, Andrew V.
;
Randolph, Peyton B.
;
Davis, Jessie R.
;
Sousa, Alexander A.
;
Koblan, Luke W.
;
Levy, Jonathan M.
;
Chen, Peter J.
;
Wilson, Christopher
;
Newby, Gregory A.
;
Raguram, Aditya
;
Liu, David R.
.
NATURE,
2019, 576 (7785)
:149-+

Anzalone, Andrew V.
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA

Randolph, Peyton B.
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA

Davis, Jessie R.
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA

Sousa, Alexander A.
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA

Koblan, Luke W.
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA

Levy, Jonathan M.
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA

Chen, Peter J.
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA

Wilson, Christopher
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA

Newby, Gregory A.
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA

论文数: 引用数:
h-index:
机构:

Liu, David R.
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
[5]
Strategies to Modulate Immune Responses: A New Frontier for Gene Therapy
[J].
Arruda, Valder R.
;
Favaro, Patricia
;
Finn, Jonathan D.
.
MOLECULAR THERAPY,
2009, 17 (09)
:1492-1503

Arruda, Valder R.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Childrens Hosp Philadelphia, Dept Pediat, Abramson Res Ctr 302F,Sch Med,Hematol Div, Philadelphia, PA 19104 USA Univ Penn, Childrens Hosp Philadelphia, Dept Pediat, Abramson Res Ctr 302F,Sch Med,Hematol Div, Philadelphia, PA 19104 USA

Favaro, Patricia
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Childrens Hosp Philadelphia, Dept Pediat, Abramson Res Ctr 302F,Sch Med,Hematol Div, Philadelphia, PA 19104 USA Univ Penn, Childrens Hosp Philadelphia, Dept Pediat, Abramson Res Ctr 302F,Sch Med,Hematol Div, Philadelphia, PA 19104 USA

Finn, Jonathan D.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Childrens Hosp Philadelphia, Dept Pediat, Abramson Res Ctr 302F,Sch Med,Hematol Div, Philadelphia, PA 19104 USA Univ Penn, Childrens Hosp Philadelphia, Dept Pediat, Abramson Res Ctr 302F,Sch Med,Hematol Div, Philadelphia, PA 19104 USA
[6]
Gene Editing on Center Stage
[J].
Bak, Rasmus O.
;
Gomez-Ospina, Natalia
;
Porteus, Matthew H.
.
TRENDS IN GENETICS,
2018, 34 (08)
:600-611

Bak, Rasmus O.
论文数: 0 引用数: 0
h-index: 0
机构:
Aarhus Univ, Aarhus Inst Adv Studies, DK-8000 Aarhus C, Denmark
Aarhus Univ, Dept Biomed, DK-8000 Aarhus C, Denmark Aarhus Univ, Aarhus Inst Adv Studies, DK-8000 Aarhus C, Denmark

Gomez-Ospina, Natalia
论文数: 0 引用数: 0
h-index: 0
机构:
Stanford Univ, Dept Pediat, Stanford, CA 94305 USA Aarhus Univ, Aarhus Inst Adv Studies, DK-8000 Aarhus C, Denmark

Porteus, Matthew H.
论文数: 0 引用数: 0
h-index: 0
机构:
Stanford Univ, Dept Pediat, Stanford, CA 94305 USA Aarhus Univ, Aarhus Inst Adv Studies, DK-8000 Aarhus C, Denmark
[7]
Gene delivery strategies for the treatment of mucopolysaccharidoses
[J].
Baldo, Guilherme
;
Giugliani, Roberto
;
Matte, Ursula
.
EXPERT OPINION ON DRUG DELIVERY,
2014, 11 (03)
:449-459

Baldo, Guilherme
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Clin Porto Alegre, Ctr Terapia Genica, BR-90035903 Porto Alegre, RS, Brazil Hosp Clin Porto Alegre, Ctr Terapia Genica, BR-90035903 Porto Alegre, RS, Brazil

Giugliani, Roberto
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Clin Porto Alegre, Ctr Terapia Genica, BR-90035903 Porto Alegre, RS, Brazil
Univ Fed Rio Grande do Sul, Programa Posgrad Genet & Biol Mol, BR-90035903 Porto Alegre, RS, Brazil Hosp Clin Porto Alegre, Ctr Terapia Genica, BR-90035903 Porto Alegre, RS, Brazil

Matte, Ursula
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Clin Porto Alegre, Ctr Terapia Genica, BR-90035903 Porto Alegre, RS, Brazil Hosp Clin Porto Alegre, Ctr Terapia Genica, BR-90035903 Porto Alegre, RS, Brazil
[8]
Treatment strategies for lysosomal storage disorders
[J].
Beck, Michael
.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY,
2018, 60 (01)
:13-18

Beck, Michael
论文数: 0 引用数: 0
h-index: 0
机构:
Johannes Gutenberg Univ Mainz, Univ Med Ctr, Inst Human Genet, Langenbeckstr 1, D-55131 Mainz, Germany Johannes Gutenberg Univ Mainz, Univ Med Ctr, Inst Human Genet, Langenbeckstr 1, D-55131 Mainz, Germany
[9]
Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis
[J].
Biasco, Luca
;
Rothe, Michael
;
Schott, Juliane W.
;
Schambach, Axel
.
HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA,
2017, 31 (05)
:737-+

Biasco, Luca
论文数: 0 引用数: 0
h-index: 0
机构:
Harvard Med Sch, Dana Farber Boston Childrens Canc & Blood Disorde, Gene Therapy Program, 1 Jimmy Fund Way, Boston, MA 02115 USA
UCL, UCL Great Ormond St Inst Child Hlth, UCL Fac Populat Hlth Sci, 30 Guilford St, London WC1N 1EH, England Harvard Med Sch, Dana Farber Boston Childrens Canc & Blood Disorde, Gene Therapy Program, 1 Jimmy Fund Way, Boston, MA 02115 USA

Rothe, Michael
论文数: 0 引用数: 0
h-index: 0
机构:
Hannover Med Sch, REBIRTH Cluster Excellence, Inst Expt Hematol, Carl Neuberg Str 1, D-30625 Hannover, Germany Harvard Med Sch, Dana Farber Boston Childrens Canc & Blood Disorde, Gene Therapy Program, 1 Jimmy Fund Way, Boston, MA 02115 USA

Schott, Juliane W.
论文数: 0 引用数: 0
h-index: 0
机构:
Hannover Med Sch, REBIRTH Cluster Excellence, Inst Expt Hematol, Carl Neuberg Str 1, D-30625 Hannover, Germany Harvard Med Sch, Dana Farber Boston Childrens Canc & Blood Disorde, Gene Therapy Program, 1 Jimmy Fund Way, Boston, MA 02115 USA

Schambach, Axel
论文数: 0 引用数: 0
h-index: 0
机构:
Hannover Med Sch, REBIRTH Cluster Excellence, Inst Expt Hematol, Carl Neuberg Str 1, D-30625 Hannover, Germany
Harvard Med Sch, Div Hematol Oncol, Boston Childrens Hosp, 300 Longwood Ave, Boston, MA 02115 USA Harvard Med Sch, Dana Farber Boston Childrens Canc & Blood Disorde, Gene Therapy Program, 1 Jimmy Fund Way, Boston, MA 02115 USA
[10]
Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications
[J].
Biffi, Alessandra
.
MOLECULAR THERAPY,
2017, 25 (05)
:1155-1162

Biffi, Alessandra
论文数: 0 引用数: 0
h-index: 0
机构:
Harvard Univ, Harvard Med Sch, Boston, MA 02115 USA
Dana Farber Boston Childrens Canc & Blood Disorde, Gene Therapy Program, Boston, MA 02115 USA
Boston Childrens Hosp, Dept Med, Program Gene Therapy Rare Dis, SM1158,450 Brookline Ave, Boston, MA 02115 USA Harvard Univ, Harvard Med Sch, Boston, MA 02115 USA