Disclosed herein are methods for treating patients that may develop or already have pre-existing gene therapy neutralizing antibodies by administering an agent that blocks, inhibits or reduces the interaction between immunoglobulin G (IgG) and the neonatal Fc receptor (FcRn), such as an anti-FcRn antibody, to reduce IgG recycling and enhance IgG clearance in vivo. Also disclosed are methods for utilizing agents that reduce interaction of IgG with FcRn for gene therapy treatment of a disease in a patient in need thereof.

Provided herein are methods of transducing a recombinant viral, e.g., retroviral, vector, improving the transgene expression from a recombinant viral vector, and reducing titers of neutralizing antibodies that bind a recombinant viral vector. In general, these methods include administering to a subject a recombinant viral vector and an effective amount of an immunosuppressive regimen.

The present disclosure relates to a polymer-iron oxide composite nanoparticle, a polymer-iron oxide composite nanoparticle including a silica coating layer coated on the surface of the polymer-iron oxide composite nanoparticle, a DNA-containing polymer-iron oxide composite nanostructure including DNA attached on the silica coating layer, a method of preparing the same and a method of controlling expression of a gene.

The present invention relates to compositions and methods, in particular to methods based on systemic injection of rAAV, for delivering genes to cells of the central nervous system in mammals, such as brain neurons or glial cells, and in particular to motor neurons or glial cells of the spinal cord The invention also relates to methods of treating motor neuron disorders in mammals by expression of therapeutic genes. The invention stems from the unexpected discovery that peripheral injection of AAV vectors leads to a bypass of the blood brain barrier and a massive infection of motor neurons. The invention may be used in any mammal, including human subjects.

A composition comprising at least one nanobomb comprising at least one first particle and at least one second particle in close proximity to the first particle. The at least one first particle is able to absorb electromagnetic radiation so as to generate a vapor bubble. The generation of the vapor bubble causes the at least one second particle to be propelled over a distance D. The composition is suitable to alter a biological barrier, in particular, for deforming, permeabilizing or perforating a biological barrier. A method to alter biological barriers is also disclosed.

Disclosed herein are molecules and pharmaceutical compositions that induce an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion. Also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that induces an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion.

Upon administration of rAAV vectors the humoral immune response (neutralizing antibodies) is the first barrier that needs to be overcome. Surprisingly it was found that by using immunoadsorption for depletion of immunoglobulins from the blood (plasma), subjects can be highly efficiently treated with rAAV vectors, i.e. obtain highly efficient transduction after rAAV vector administration, in spite of the presence of high levels of nAb.

The present disclosure teaches methods of treating a patient who has an advanced metastatic cancer, after the patient has the patient has failed at least one treatment regimen for the advanced metastatic cancer, by administering a plurality of infusions of a vector comprising a tumor signature-targeting peptide and a nucleic acid that encodes a dominant negative human cyclin G1 construct. One or more of the patient's treatment regimens may have included gemcitabine. The present disclosure also provides methods of treatment by further administering to the patient an additional therapeutic agent such as an immune-modulatory monoclonal antibody, a cytotoxic chemotherapeutic agent, an anti-angiogenesis agent, a selective tyrosine kinase inhibitor, or a monoclonal antibody directed against specific features of cells from the metastatic cancer.

The present invention provides a method for treating an auditory neuropathy spectrum disorder in a subject comprising transferring the gene of DFNB59 via an adeno-associated virus (AAV) vector to the subject.

The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.