Described herein are compositions comprising AAV vectors comprising a sequence encoding mucolipin 1, and methods of use thereof for gene therapy of Mucolipidosis IV (MLIV).

Compositions, non-viral vectors, recombinant viruses, and recombinant viral vectors for inhibiting ΔFosB expression or activity in a cell and for treating dyskinesia in a subject (e.g., a human patient having Parkinson's disease and Levodopa-induced dyskinesia) include a nucleic acid sequence encoding a shRNA specific for ΔFosB. Methods of using these compositions, non-viral vectors, recombinant viruses, and recombinant viral vectors are also described herein. These compositions, non-viral vectors, recombinant viruses, and recombinant viral vectors and methods of use provide novel therapies for dyskinesia based on the reduction of ΔFosB expression and/or activity.

Compositions, non-viral vectors, recombinant viruses, and recombinant viral vectors for inhibiting ΔFosB expression or activity in a cell and for treating dyskinesia in a subject (e.g., a human patient having Parkinson's disease and Levodopa-induced dyskinesia) include a nucleic acid sequence encoding a shRNA specific for ΔFosB. Methods of using these compositions, non-viral vectors, recombinant viruses, and recombinant viral vectors are also described herein. These compositions, non-viral vectors, recombinant viruses, and recombinant viral vectors and methods of use provide novel therapies for dyskinesia based on the reduction of ΔFosB expression and/or activity.

The present disclosure provides methods and compositions for genetically modifying hematopoietic stem and progenitor cells (HSPCs), in particular by replacing the HBA1 or HBA2 locus in the HSPCs with a transgene encoding a therapeutic protein.

The present disclosure provides methods and compositions for genetically modifying hematopoietic stem and progenitor cells (HSPCs), in particular by replacing the HBA1 or HBA2 locus in the HSPCs with a transgene encoding a therapeutic protein.

The present invention is directed to heterodimeric anti-LAG-3×anti-CTLA-4. Also provided are nucleic acid compositions that encode the antibodies, expression vector compositions that include the nucleic acids, and host cells that include the expression vector compositions.

The disclosure pertains to a recombinant adeno-associated virus (rAAV) comprising an Anc80L65 capsid for delivering a polynucleotide (e.g., a transgene) into the central nervous system (CNS). Further provided includes methods for treating CNS diseases using the rAAV and pharmaceutical compositions comprising the rAAV.

The disclosure pertains to a recombinant adeno-associated virus (rAAV) comprising an Anc80L65 capsid for delivering a polynucleotide (e.g., a transgene) into the central nervous system (CNS). Further provided includes methods for treating CNS diseases using the rAAV and pharmaceutical compositions comprising the rAAV.

An object of the present invention is to provide a method of treating cancer using a checkpoint inhibitor in combination with REIC/Dkk-3 gene. The present invention is a combination pharmaceutical kit for treating cancer comprising REIC/Dkk-3 in combination with a check point inhibitor and a method for treating cancer by administering REIC/Dkk-3 gene and a check point inhibitor to a cancer patient.

An object of the present invention is to provide a method of treating cancer using a checkpoint inhibitor in combination with REIC/Dkk-3 gene. The present invention is a combination pharmaceutical kit for treating cancer comprising REIC/Dkk-3 in combination with a check point inhibitor and a method for treating cancer by administering REIC/Dkk-3 gene and a check point inhibitor to a cancer patient.