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7 Μαΐ 2024 · Important safety concerns include immune reactions to the vector, the foreign DNA or the new protein resulting from gene therapy, and a remaining low cancer risk based on insertional...
26 Νοε 2015 · This book provides detailed, up-to-date topics addressing basic principles of gene therapy and discussing some of the challenges encountered by scientists in developing this relatively novel technology.
1 Σεπ 2017 · Gene therapy: advances, challenges and perspectives. September 2017. Einstein (São Paulo 15 (3):369-375. DOI: 10.1590/s1679-45082017rb4024. License. CC BY. Authors: Giulliana Augusta Rangel...
8 Νοε 2023 · In this article, we highlight success and ongoing challenges in three areas of high activity in gene therapy: inherited blood cell diseases by targeting hematopoietic stem cells, malignant...
The chapters in this book cover a range of strategies for molecular and cellular therapies for human disease, their advantages, and central challenges to their widespread application. Potential solutions to these issues are also discussed in detail.
7 Μαΐ 2024 · To address this knowledge gap, this review summarizes core principles, benefits, potential risks and challenges of gene therapy, with a particular focus on hemophilia and spinal muscular atrophy, and discusses future perspectives.
1 Ιαν 2011 · Gene therapy is a clinical therapeutic procedure in which genes are intentionally introduced into human somatic cells for turning on or restoring a gene function (gain of function) to produce a therapeutic protein with the desired action.
