Wiskott-Aldrich Syndrome Therapeutics - Pipeline Analysis 2018
by Manish Kumar Digital AnalystWiskott-Aldrich syndrome is a hereditary immunodeficiency disorder
characterized by abnormal functioning of immune system and decreased ability to
form blood clots. The abnormality in the platelets leads to easy bruising
effects in case of prolonged bleeding.
The white blood cells become non-functional and cause other immunological
disorders in Wiskott-Aldrich syndrome. It has been discovered that this disease
is caused by the mutation in WAS gene (Xp11.4-p11.21) that instruct to make
Wiskott-Aldrich syndrome protein.
Some of the symptoms observed in the patients are thrombocytopenia, hematologic abnormalities, eczema, and malignancies. Genethon Inc. is in the process of developing autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene as a gene therapy for the treatment of Wiskott-Aldrich syndrome. Further, the University of Pittsburgh and Orchard Therapeutics Limited are also involved in the pipeline for Wiskott-Aldrich syndrome.
Get the detailed analysis @ https://www.pharmaproff.com/report/wiskott-aldrich-syndrome-therapeutics-pipeline-analysis
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Created on May 8th 2019 08:24. Viewed 390 times.