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Therapy: Gene Therapy
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Total 388 results found since Jan 2013.
Organ function indications and potential improvements following curative therapy for sickle cell disease
Hematology Am Soc Hematol Educ Program. 2022 Dec 9;2022(1):277-282. doi: 10.1182/hematology.2022000372.ABSTRACTCurative therapies for sickle cell disease include allogeneic hematopoietic stem cell transplantation (HSCT) and gene-modified autologous stem cell transplantation. HSCT has been used for 30 years with success measured by engraftment, symptom control, graft-vs-host disease (GVHD) risk, organ toxicity, and immune reconstitution. While human leukocyte antigen-matched sibling donor (MSD) transplants have excellent outcomes, alternate donor transplants (unrelated/haploidentical) are just beginning to overcome GVHD and...
Source: Hematology ASH Education Program - December 9, 2022 Category: Hematology Authors: Monica L Hulbert Allison A King Shalini Shenoy Source Type: research
Heterogeneity of the long-term economic burden of severe sickle cell disease: a 5-year longitudinal analysis
CONCLUSION: Results indicate high clinical need and economic burden among patients with severe presentation of SCD. These findings not only highlight the need for improved therapeutic options to limit or prevent disease progression, but also starts to provide insight on lifetime costs of SCD that will be needed in the evaluation of emerging curative intent therapies.PMID:36222016 | DOI:10.1080/13696998.2022.2133824
Source: Journal of Medical Economics - October 12, 2022 Category: Health Management Authors: Meghan Gallagher Anjulika Chawla Brenna L Brady Sherif M Badawy Source Type: research
Advanced molecular therapies for neurological diseases: focus on stroke, alzheimer's disease, and parkinson's disease
AbstractNeurological diseases (NDs) are one of the leading causes of disability and the second leading cause of death globally. Among these stroke, Alzheimer's disease (AD), and Parkinson's disease (PD) are the most common NDs. A rise in the absolute number of individuals affected with these diseases indicates that the current treatment strategies in management and prevention of these debilitating diseases are not effective sufficiently. Therefore, novel treatment strategies are being explored to cure these diseases by addressing the causative mechanisms at the molecular level. Advanced therapies like gene therapy (gene ed...
Source: Neurological Sciences - September 6, 2022 Category: Neurology Source Type: research
Genetics of Dravet Syndrome and its Targeted Therapy by Nanomedicine: A Roadmap for Future Treatment of Drug Resistant Seizures
Curr Mol Pharmacol. 2022 Aug 19. doi: 10.2174/1874467215666220819143105. Online ahead of print.ABSTRACTAccording to the World Health Organization (WHO), epilepsy is the 4th most prevalent neurological disorder after migraine, stroke, and Alzheimer's disease. There are numerous types of epileptic syndrome that are reported in children; one of them is Dravet syndrome. It is a neurological disorder of infants' outset during the first year of life. Dravet syndrome is a genetically determined syndrome and the most studied form of genetic epilepsy. Nearly 70-80% of its cases are due to genetic alterations in the SCN1A gene, and ...
Source: Epilepsy Curr - August 20, 2022 Category: Neurology Authors: Muhammad Ikram Sufian Rasheed Source Type: research
