Open Access
snRNA progenitor cells following lentiviral vector delivery of an antisense High-level expression of hemoglobin A in human thalassemic erythroid
Marla M. Vacek,Hong Ma,Federica Gemignani,Giuseppina Lacerra,Tal Kafri,Ryszard Kole +5 more
- 01 Jan 2013
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TL;DR: This paper aims to provide a history ofGene therapy in the clinic and some of the techniques used have been described in detail in a number of clinics around the world.
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Abstract: http://bloodjournal.hematologylibrary.org/content/101/1/104.full.html Updated information and services can be found at: (517 articles) Gene Therapy Articles on similar topics can be found in the following Blood collections http://bloodjournal.hematologylibrary.org/site/misc/rights.xhtml#repub_requests Information about reproducing this article in parts or in its entirety may be found online at: http://bloodjournal.hematologylibrary.org/site/misc/rights.xhtml#reprints Information about ordering reprints may be found online at: http://bloodjournal.hematologylibrary.org/site/subscriptions/index.xhtml Information about subscriptions and ASH membership may be found online at:
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References
In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector
Luigi Naldini,Ulrike Blömer,Philippe Gallay,Daniel S. Ory,Richard C. Mulligan,Fred H. Gage,Inder M. Verma,Didier Trono +7 more
TL;DR: The ability of HIV-based viral vectors to deliver genes in vivo into nondividing cells could increase the applicability of retroviral vectors in human gene therapy.
5.4K
•Book
Hematology: Basic Principles and Practice
Ronald Hoffman
- 01 Apr 1995
TL;DR: The author examines the immune system through the lens of Epstein-Barr Virus-Associated Diseases, as well as the biology of Stem Cells and Disorders of Hematopoiesis, and the approach to the Adult and Child with Anemia.
1.9K
Exonic splicing enhancers: mechanism of action, diversity and role in human genetic diseases
TL;DR: This work has suggested that many human genetic diseases linked to mutations within exons might be caused by the inactivation of ESEs, and suggested a new model for ESE function.
772
Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
Chad May,Stefano Rivella,John Callegari,Glenn Heller,Karen M. L. Gaensler,Lucio Luzzatto,Michel Sadelain +6 more
TL;DR: It is shown that the use of recombinant lentiviruses enables efficient transfer and faithful integration of the human β-globin gene together with large segments of its locus control region, which should be of therapeutic benefit in patients with severe defects in haemoglobin production.
587
Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.
Zbigniew Dominski,Ryszard Kole +1 more
TL;DR: These experiments represent an approach in which antisense oligonucleotides are used to restore the function of a defective gene and not, as usual, to down-regulate the expression of an undesirable gene.
452
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