Patent Application Titled "Enhancers And Vectors" Published Online (USPTO 20240287546).

The patent application titled "Enhancers and Vectors" discusses the use of gene therapy to treat disorders associated with defective WAS protein expression, such as Wiskott-Aldrich Syndrome (WAS). The application aims to develop more therapies for these disorders, particularly gene therapies that do not rely on suitable donors. It provides detailed information on the nucleic acid sequences and compositions involved in the gene therapy approach, including recombinant vectors that can be used to treat diseases associated with deficient expression of a specific gene product. The application also describes methods of treating diseases like Wiskott-Aldrich Syndrome by transducing stem cells or progenitor cells with the vectors and transplanting them into the patient. [Extracted from the article]