Annotated Bibliography on "Gene Therapy Treatment in Sickle Cell Disease"

Annotated Bibliography 4 pages (1128 words) Sources: 7

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Sickle Gene

Gene Therapies for Sickle Cell Anemia: Annotated Bibliography

Kohn, D. & Candotti, F. (2009). Gene therapy fulfilling its promise. NEJM 360(5): 518-21. This brief and relatively broad review details a diversity of the advances that have been made in gene therapy since techniques first began to emerge in the latter half of the twentieth century. A wide variety of different disorders and diseases have been treated and relived if not put into full remission/disappearance through the use of gene therapies, and though such therapies are not the magic bullet that the public often sees them as they do represent a more effective means of addressing many diseases than has been heretofore available. The author focuses on emerging treatments for X-linked SCID

Lucarelli G., Gaziev, J., Isgro, a., Sodani, P…. & Andreani, M. (2012). Allogeneic cellular gene therapy in hemoglobinopathies -- evaluation of hematopoietic SCT in sickle cell anemia. Bone Marrow Transplantation 47: 227-30.

This study examined eleven patients suffering from sickle cell anemia and tracked their progress following treatment with stem cell transplantation from an HLA-identical and related donor. One patient in the study died a year following the transplantation, however the remaining ten patients were still free of any sickle cell anemia symptoms five years following the transplantation. This research confirms that stem cell transplantation from suitable HLA-identical donors is a promising therapy that provides an excellent survival rate and the ability for patients to lead sickle cell-free lives.

Morstyn, G. & Sheridan, W. (2006). Cell Therapy. New York: Cambridge University Press.

This textbook contains broad yet advanced descriptions of stem cell and certain other genetic therapy techniques that developed in the last several decade providing a state-of-the-art glimpse into certain practices and capabilities. The authors do not have a specific perspective or viewpoint that they attempt to assert nor do they engage in any primary research themselves, but instead they provide an overview of the science of cell therapy and many of its specific incarnations. While not directly related to sickle cell anemia (at least not wholly so), the information this source contains on cell therapies is highly relevant and useful for foundational knowledge.

Papanikolaou, E. & Anagnou, N. (2010). Major Challenges for Gene Therapy of Thalassemia and Sickle Cell Disease. Current Gene Therapy 10(5): 404-12. Retroviral vectors have been postulated as potentially useful agents in certain gene therapies, yet a variety of problems exist in their use that have hindered research and practical applications. Inefficient gene transfer times and capabilities are noted as especially problematic in retroviral vector use, and these researchers explore other potential vectors noting the specific opportunities and risks or problems of each of the alternatives they identify. No recommendations for specific therapeutic applications are made, however an abundance of theoretical and research-applicable information is provided that makes the study quite worthwhile.

Ray, a., Pearlman, M., Vats, T. & Khatua, S. (2011). Development of ependymoma following allogeneic cord blood transplantation in a sickle cell patient. Neuro-Oncology 13(3): 95-101.

This case study examines the risk of tumor development following the use of cord-blood gene therapies to treat sickle cell anemia following the presentation of a sixteen-year-old male patient with headache and ataxia and found to have a tumor in the posterior fossa. The patient had received a cord blood transfusion at the age of ten, and this is the only instance the authors were aware of in… READ MORE

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