Wiskott-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.
Request
to Get the Sample Pages at: https://www.pharmaproff.com/request-sample/1152
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.
The report
provides a comprehensive understanding of the pipeline activities covering all
drug candidates under various stages of development, with the detailed analysis
of pipeline and clinical trials.
Pipeline
analysis of drugs by phases includes product description and development
activities including information about clinical results, designations,
collaborations, licensing, grants, technology, and others.
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