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Gene Therapy Basics
What Are the Recent Gene Therapy Studies and Trials for Sickle Cell Disease?
27 resources available:
Myelodysplastic Syndrome Unrelated to Lentiviral Vector in a Patient Treated With Gene Therapy for Sickle Cell Disease
Source: Blood Advances Year: 2020Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease
Source: Current Gene Therapy Year: 2015Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene
Source: Human Gene Therapy Year: 2016Gene Therapy in a Patient With Sickle Cell Disease
Source: The New England Journal of Medicine Year: 2017Genetic Therapies in Sickle Cell Disease
Source: National Heart, Lung, and Blood Institute (NHLBI) Year: 2019A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
Source: ClinicalTrials.gov (bluebird bio) Year: 2021Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients With Sickle Cell Disease
Source: ClinicalTrials.gov (Assistance Publique - Hôpitaux de Paris) Year: 2021Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy
Source: ClinicalTrials.gov (bluebird bio) Year: 2021Study of Safety and Efficacy of Genome - Edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)
Source: ClinicalTrials.gov (Novartis Pharmaceuticals) Year: 2021CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
Source: The New England Journal of Medicine Year: 20211st CRISPR Gene Editing Trial Slated to Open in Severe SCD Patients
Source: BioNews, Inc. Year: 2021CRISPR and Another Genetic Strategy Fix Cell Defects in Two Common Blood Disorders
Source: Science | AAAS Year: 2020Current Hot Topics and Emerging Trends in Gene Therapy
Source: The Alliance for Regenerative Medicine (ARM) Year: 2021Cell and Gene Therapy: Overview, Current Landscape and Future Trends
Source: The Journal of Precision Medicine Year: N/AGene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease (CEDAR)
Source: ClinicalTrials.gov (Graphite Bio, Inc.) Year: 2021Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
Source: ClinicalTrials.gov (Mark Walters, MD) Year: 2021A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
Source: ClinicalTrials.gov (Vertex Pharmaceuticals Incorporated) Year: 2021A Study of IMR-687 in Subjects With Sickle Cell Disease
Source: ClinicalTrials.gov (Imara, Inc.) Year: 2021A Long-term Follow-Up Study in Subjects Who Received CTX001
Source: ClinicalTrials.gov (Vertex Pharmaceuticals Incorporated) Year: 2021New Gene-Editing Technique Shows Promise Against Sickle Cell Disease
Source: The Harvard Gazette - Harvard University Year: 2021Study of Safety and Efficacy of Genome-Edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)
Source: ClinicalTrials.gov (Novartis Pharmaceuticals) Year: 2021To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
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