دورية أكاديمية
أعرض في EDS

Advancements in Hematopoietic Stem Cell Gene Therapy: A Journey of Progress for Viral Transduction.

التفاصيل البيبلوغرافية
العنوان: Advancements in Hematopoietic Stem Cell Gene Therapy: A Journey of Progress for Viral Transduction.
المؤلفون: Giommetti A; Miltenyi Biotec B.V. & Co. KG, 51429 Bergisch Gladbach, Germany.; Faculty of Biology, University of Freiburg, 79104 Freiburg, Germany., Papanikolaou E; Miltenyi Biotec B.V. & Co. KG, 51429 Bergisch Gladbach, Germany.; Laboratory of Biology, School of Medicine, National and Kapodistrian University of Athens, 115 27 Athens, Greece.
المصدر: Cells [Cells] 2024 Jun 15; Vol. 13 (12). Date of Electronic Publication: 2024 Jun 15.
نوع المنشور: Journal Article; Review
اللغة: English
بيانات الدورية: Publisher: MDPI Country of Publication: Switzerland NLM ID: 101600052 Publication Model: Electronic Cited Medium: Internet ISSN: 2073-4409 (Electronic) Linking ISSN: 20734409 NLM ISO Abbreviation: Cells Subsets: MEDLINE
أسماء مطبوعة: Original Publication: Basel, Switzerland : MDPI
مواضيع طبية MeSH: Genetic Therapy*/methods , Hematopoietic Stem Cells*/metabolism , Transduction, Genetic* , Genetic Vectors*, Humans ; Animals ; Hematopoietic Stem Cell Transplantation ; Gene Editing/methods
مستخلص: Hematopoietic stem cell (HSC) transduction has undergone remarkable advancements in recent years, revolutionizing the landscape of gene therapy specifically for inherited hematologic disorders. The evolution of viral vector-based transduction technologies, including retroviral and lentiviral vectors, has significantly enhanced the efficiency and specificity of gene delivery to HSCs. Additionally, the emergence of small molecules acting as transduction enhancers has addressed critical barriers in HSC transduction, unlocking new possibilities for therapeutic intervention. Furthermore, the advent of gene editing technologies, notably CRISPR-Cas9, has empowered precise genome modification in HSCs, paving the way for targeted gene correction. These striking progresses have led to the clinical approval of medicinal products based on engineered HSCs with impressive therapeutic benefits for patients. This review provides a comprehensive overview of the collective progress in HSC transduction via viral vectors for gene therapy with a specific focus on transduction enhancers, highlighting the latest key developments, challenges, and future directions towards personalized and curative treatments.
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معلومات مُعتمدة: 101072427 European Union
فهرسة مساهمة: Keywords: gene therapy; hematopoietic stem cells (HSC); rare diseases; transduction; transduction enhancers; viral vectors
تواريخ الأحداث: Date Created: 20240626 Date Completed: 20240626 Latest Revision: 20240628
رمز التحديث: 20240628
مُعرف محوري في PubMed: PMC11201829
DOI: 10.3390/cells13121039
PMID: 38920667
قاعدة البيانات: MEDLINE
الوصف
تدمد:2073-4409
DOI:10.3390/cells13121039