The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Mller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.

The present invention relates to nucleic acid expression cassettes and vectors containing liver-specific regulatory elements and codon-optimized factor IX or factor VIII transgenes, methods employing these expression cassettes and vectors and uses thereof. The present invention is particularly useful for applications using liver-directed gene therapy, in particular for the treatment of hemophilia A and B.

A recombinant mutant BoV genome is provided, as well as methods of using the vector, e.g., to prepare helper-free virus.

Described is an adenovirus derived helper virus which may comprise the adenoviral DNA sequences for E2a, S4 (orf6), the VA1 RNA gene, and the parvovirus VP capsid gene unit. Also described is a method of efficiently preparing a recombinant parvovirus (particle) which is based on the use of various adenoviral derived helper viruses/vectors.

Methods for delivering a therapeutic agent to a cerebral cortex or to the cerebral cortex and spinal cord include administering a viral vector that includes the therapeutic agent into white matter of a brain in order to deliver the therapeutic agent to the cerebral cortex or to the cerebral cortex and spinal cord. Kits for performing such methods may include at least one viral vector, at least one therapeutic agent, and a convection enhanced delivery (CED) catheter.

The invention relates to a method for the treatment of amyotrophic lateral sclerosis (ALS). Specifically, the invention implements the use of an antisense sequence adapted to affect alternative splicing in a human SOD1 pre-mRNA, thereby leading to the destruction of the skipped m RNA by the cell machinery.

Various aspects of the invention provide for capsids, parvovirus capsids, hybrid parvovirus capsids, parvovirus vectors, hybrid parvovirus vectors, hybrid parvovirus particles and parvovirus particles containing polypeptides in which the sequence YCDGFYACYMDV (SEQ ID NO: 3) has been substituted into the VP2 loop of the B19 capsid protein. Polypeptides in which the sequence YCDGFYACYMDV (SEQ ID NO: 3) has been substituted into the VP2 loop of the B19 capsid protein are also provided (e.g., SEQ ID NO: 2). Other aspects of the invention provides capsids, parvovirus capsids, hybrid parvovirus capsids, parvovirus vectors, hybrid parvovirus vectors, hybrid parvovirus particles and parvovirus particles containing a polypeptide comprising SEQ ID NO: 2. Also provided in various aspects of the invention a pharmaceutical compositions and methods of delivering therapeutic agents and/or reporter peptides/proteins to target cells. Finally, methods of treating diseases characterized by cells expressing HER2/neu receptors are also provided.

Described is an adenovirus derived helper virus which may comprise the adenoviral DNA sequences for E2a, S4 (orf6), the VA1 RNA gene, and the parvovirus VP capsid gene unit. Also described is a method of efficiently preparing a recombinant parvovirus (particle) which is based on the use of various adenoviral derived helper viruses/vectors.

The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 200 bp having at least 70% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in cones and/or OFF bipolar cells of a gene when operatively linked to a nucleic acid sequence coding for said gene.

Described herein are compositions and kits comprising recombinant adeno-associated viruses (rAAVs) with increased M transduction enrichment in the heart. The rAAV compositions described herein encapsidate a transgene, such as a therapeutic nucleic acid. Gene therapy using the rAAVs is described. Also described are methods of treating cardiovascular-related diseases and conditions.