Clinical Presentation of Genetic Disorders in Patients Attending Genetic Outpatient Clinic of Assiut University Children Hospital

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ClinicalTrials.gov Identifier: NCT05888155

Recruitment Status : Not yet recruiting

First Posted : June 5, 2023

Last Update Posted : June 5, 2023

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View this study on the modernized ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Shaimaa Ali Soliman Mohammed, Assiut University

No Study Results Posted on ClinicalTrials.gov for this Study

About Study Results Reporting on ClinicalTrials.gov

Recruitment Status :	Not yet recruiting
Estimated Primary Completion Date :	August 2024
Estimated Study Completion Date :	September 2024

Publications of Results:

National Institute of General Medical Sciences (301_496_7301): Supports basic research that increase understanding of biological processes&lays the foundation for advances in disease diagnosis, treatment&prevention.

Other Publications:

MidlinePlus.gov:Medlineplus(internet) .Bethesda (MD)National library of medicine (US)(updated june24)cited (2020Jul 1.)

Cavazzano human beta-- Calvo, M. (2010). Transfusion thalassaemia. Nature 467, 318independence and HMGA2 activation after gene therapy of 322. doi:10.1038/nature09328 Cideciyan, A.V. et al (22 January 2013). Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal de generation despite enduring visual improvement. Proceedings of the National Academy of Sciences of the United States of America, Earline Online Publication. doi: 10.1073/pnas.1218933110 MacLaren, R.E. et al (16 January 2014). Retinal gene therapy in patien ts with choroideremia: initial findings from a phase 1/2 clinical trial. The Lancet, Early Online Publication. doi:10.1016/S01406736(13)62117 Nathwani, A.C. (2011). Adenovirusassociated virus vector-- 0 mediated gene transfer in hemophilia B. The New Engla nd Journal of Medicine, 365(25), 23572365. Nienhuis, A.W. (2013). Development of gene therapy for blood disorders: an update. Blood 122(9), 1556 1564. doi: 10.1182/blood201304453209 Palfi, S. et al (10 January 2014). Longterm safety and tolerability o f ProSavin, a lentiviral vector-- based gene therapy for parkinson's disease; a dose escalation, open Publication. doi: 10.1016/S014006736(13)61939Xlabel, phase 1/2 trial. The Lancet, Early Online Penn Medicine (7 December 2013). Penn medicine team reports findings from research study of first 59 adult and pediatric leukemia patients who received investigational, personalized cellular therapy CTL019. Retrieved from http://www.uphs.upenn.edu/news/News_Releases/2013/12/ctl019/ Persons, Derek A. (2010). Gene t 10.1038/467277a Petrsherapy: Targeting betathalassaemia. Nature 467, 277278. doi: Silva, H. & R. Linden (2014). Advances in gene therapy technologies to treat retinitis pigmentosa.

3)National Human genome Research Institute; Led by Director Eric Green, M.D., Ph.D., the National Human Genome Research Institute (NHGRI) is the driving force for advancing genomics research at the National Institutes of Health (NIH), the largest biomedical research agency in the world. (18/5/2018)

Garvan Institute of Medical Research/an Australian biomedical research institute located in Darlinghurst, Sydney, New South Wales/ directed by professor Chris Goodnow , FAA, FRS.

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