In vitro and in vivo enrichment strategy targeting lymphocytes derived from vector transduced HSCs for therapy of disorders

A dual promoter lentivirus vector that expresses 1) a protein of interest, wherein the protein of interest is encoded by a transgene and wherein the protein of interest is associated with a lysosomal storage disorder and 2) a mutant form of inosine-5′-monophosphate dehydrogenase 2 (IMPDH2(IY)), wherein the lentivirus vector when transduced into a host cell confers resistance to mycophenolic acid (MPA) and/or mycophenolate mofetil (MMF) in vitro and/or in vivo and, wherein the mutant form of IMPDH2(IY) is encoded by the nucleotide sequence of SEQ ID NO:

• 7, or a sequence having at least 85% sequence identity to the nucleotide sequence of SEQ ID NO:
• 7.