Intrabiliary hydrodynamic injection of nucleic acid for in vivo gene therapy for treatment of liver or pancreas disease and other disorders.

The present disclosure provides a method of restoring or enhancing visual function in an individual, the method comprising administering to the individual a nucleic acid comprising a nucleotide sequence encoding one or more of a medium wavelength cone opsin (MW-opsin), a long wavelength cone opsin (LW-opsin), and a short wavelength cone opsin (SW-opsin). One or more of the MW-opsin, LW-opsin, and SW-opsin is expressed in a retinal cell in the individual, thereby restoring or enhancing visual function.

Provided is an enveloped viral vector comprising a viral particle surrounded by an envelope, wherein the viral particle comprises a heterologous transgene, and the envelope comprises a lipid bilayer and one or more immunosuppressive molecules, and methods for preparing and using same.

The disclosure relates to compositions and methods for treatment of diseases associated with aberrant lysosomal function, such as fronto-temporal dementia (FTD). The disclosure also provides expression constructs comprising a transgene encoding progranulin or a portion thereof. The disclosure provides methods of treating FTD by administering such expression constructs to a subject in need thereof.

A method of treating a congenital birth defect includes detecting the presence of at least one mutated gene associated with a birth defect and injecting foreign genetic material containing at least one non-mutated version of the detected mutated gene into a patient, thereby promoting a desired therapeutic outcome in the patient. A kit for treating a congenital birth defect in a patient comprising normal genetic material tailored to treat the congenital birth defect and an injection device for providing the normal genetic material to the patient is also described.

A method of treating cancerous tumors is presented herein. The method includes injecting an effective dose of a plasmid encoded for IL-12, B7-1 or IL-15 into a cancerous tumor and subsequently administering at least one high voltage, short duration pulse to the tumor. The electroporation pulses may be administered at al least 700 V/cm for a duration of less than 1 millisecond. The intratumor treatments with electroporation may be administered in at least a two-treatment protocol with the time between treatments being about 7 days. The intratumor treatments with electroporation may be administered in a three-treatment protocol with a time of four days between the first and second treatments and a time of three days between the second and third treatments. It was found that the intratumor treatments using electroporation not only resulted in tumor regression but also induced an immune memory response which prevented the formation of new tumors.

The present invention relates to an adeno-associated virus (AAV), comprising an insertion of at least 6-8 amino acids between the positions corresponding to position 587 and 588 of SEQ ID NO: 1. Also envisioned are AAVs of the present invention for use as a medicament and pharmaceutical compositions comprising the AAV of the present invention. Further, the present invention relates to an in vitro use of AAV of the present invention for transduction of the nucleus of retinal cells. Also concerned is a method for screening an insertion sequence as well as a peptide obtainable by the method for screening. Also contemplated are kits comprising the AAV of the present invention.

Provided are methods of treating an ocular disease or disorder in a subject, comprising: administering a unit dose of a pharmaceutical composition to a first eye the subject via intravitreal (IVT) injection at a first time point, and administering a second unit dose of the pharmaceutical composition to a contralateral eye of the subject via IVT injection at a second time point.

Provided herein are recombinant adeno-associated virus (rAAV) vectors comprising nucleic acid encoding N-acetyl-glucosamine-1-phosphate transferase, alpha and beta subunits (GNPTAB) and at least one AAV inverted terminal repeat (ITR). In some embodiments, the rAAV vectors may be included in a rAAV particle, which may be contained in pharmaceutical compositions and kits. These vectors, particles, compositions, and kits may find use, inter alia, in methods and uses related to treating mucolipidosis type II (ML II) or mucolipidosis type III (ML III) in a mammal, or related to increasing body size, bone mineral content, and/or bone mineral density in a mammal with mucolipidosis type II (ML II) or mucolipidosis type III (ML III).

The present disclosure provides methods of treating a human having a disease or disorder that would benefit from increasing platelet counts. The method involves inhibiting the enzyme activity of biliverdin IX reductase (BLVRB) activity or inhibiting the expression of BLVRB gene.