In the field of
gene therapy, a major hurdle is the design and identification of constructs and
gene therapy vectors providing therapeutic effects while displaying satisfactory safety profiles. In the treatment of
Diamond-Blackfan
Anemia (DBA), therapies alleviating several crucial
anemia symptoms, such as blood or
bone marrow cellularity,
hemoglobin levels, erythrocytes levels, or
platelet levels, while showing satisfactory safety profiles remain a challenge. The present invention provides constructs encoding
ribosomal protein genes involved in DBA, such as genes encoding RPS19, RPS17, RPS24, RPS10, RPL35a, RPL11, RPS26, and RPL5, vectors, methods, cells, and medical uses thereof, addressing these challenges and finding particular applications in the field of
autologous cell therapy treatment of DBA. Further, the present invention provides a non-genotoxic conditioning protocol for preparing a subject prior to
cell therapy treatment for DBA using construct of the present invention.