A systems pharmacology model for gene therapy in sickle cell disease-Reference-Cited by-同舟云学术

A systems pharmacology model for gene therapy in sickle cell disease

Published:2021-06-17 Issue:7 Volume:10 Page:696-708
ISSN:2163-8306
Container-title:CPT: Pharmacometrics & Systems Pharmacology
language:en
Short-container-title:CPT Pharmacometrics Syst Pharmacol

Author:

Zheng Bo¹,Wille Lucia²,Peppel Karsten¹,Hagen David²^ORCID,Matteson Andrew²,Ahlers Jeffrey¹,Schaff James²,Hua Fei²,Yuraszeck Theresa¹²,Cobbina Enoch¹^ORCID,Apgar Joshua F.²^ORCID,Burke John M.²,Roberts John¹,Das Raibatak²

Affiliation:

1. CSL Behring King of Prussia Pennsylvania USA

2. Applied BioMath LLC Concord Massachusetts USA

Funder

CSL Behring

Publisher

Wiley

Subject

Pharmacology (medical),Modelling and Simulation

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/psp4.12638

Reference53 articles.

1. Sickle cell disease

2. Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-GlobinGene

3. Management of Sickle Cell Disease

4. Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease

5. A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction

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