Current and Future Perspective in Hematopoietic Stem Progenitor Cell-gene Therapy for Inborn Errors of Metabolism-Reference-Cited by-同舟云学术

登录注册会员服务联系我们

Current and Future Perspective in Hematopoietic Stem Progenitor Cell-gene Therapy for Inborn Errors of Metabolism

Published:2023-09-12 Issue:10 Volume:7 Page:e953
ISSN:2572-9241
Container-title:HemaSphere
language:en
Short-container-title:

Author:

Tucci Francesca¹²,Consiglieri Giulia¹²³,Cossutta Matilde¹⁴,Bernardo Maria Ester¹²⁵

Affiliation:

1. Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Milan, Italy

2. San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy

3. Department of Biomedicine and Prevention, University of Rome Tor Vergata, Italy

4. University of Rome Tor Vergata, Italy

5. “Vita-Salute” San Raffaele University, Milan, Italy

Publisher

Wiley

Subject

Hematology

Reference59 articles.

1. Inborn errors of metabolism.;Ezgu;Adv Clin Chem,2016

2. Lysosomal storage diseases--the horizon expands.;Boustany;Nat Rev Neurol,2013

3. Substrate reduction therapy.;Platt;Acta Paediatr,2008

4. Therapeutic approaches in lysosomal storage diseases.;Fernandez-Pereira;Biomolecules,2021

5. The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders.;Broomfield;J Inherit Metab Dis,2016

Cited by 6 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. A GLB1 transgene with enhanced therapeutic potential for the preclinical development of ex-vivo gene therapy to treat mucopolysaccharidosis type IVB;Molecular Therapy - Methods & Clinical Development;2024-09

2. Systematic Review of Genetic Substrate Reduction Therapy in Lysosomal Storage Diseases: Opportunities, Challenges and Delivery Systems;BioDrugs;2024-08-23

3. Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency;Molecular Therapy;2024-08

4. TALEN-mediated intron editing of HSPCs enables transgene expression restricted to the myeloid lineage;Molecular Therapy;2024-06

5. Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of Multiple Sulfatase Deficiency;2024-03-12

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线，采集、加工和组织学术论文而形成的新型学术文献查询和分析系统，可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容，当前同舟云学术共收录了国内外主流学术期刊6万余种，收集的期刊论文及会议论文总量共计约1.5亿篇，并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询！咨询电话：010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号京ICP备18003416号-3