Present application relates to a strategy for compensating for transesterification degradation of lipid-encapsulated RNA, such as mRNA-LNP, in liquid formulations for high-volume distribution. This involves determining the rate of degradation of the encapsulated RNA and calculating an appropriate overage relative to the intended dose. Alternatively, a higher dose of the RNA may be administered to compensate for loss of effective RNA or the RNA may be formulated in higher purity in anticipation of degradation. The strategy provides a balance between supplying effective and safe products and the need for costly manufacturing processes or transportation hurdles, such as cold-chain supply.

Provided herein are methods and related compositions or kits for administering viral transfer vectors in combination with synthetic nanocarriers comprising an immunosuppressant and an anti-IgM agent.

The present disclosure relates to an anti-miRNA delivery system, and more specifically, relates to a technique of using a cancer-targeting anti-miRNA delivery system including porous silicon nanoparticles containing anti-miRNA to which a cancer cell surface protein-binding peptide is conjugated, for use in treating cancer. As a result of intensive studies in order to use and apply anti-miR-21 oligonucleotides to the treatment of ovarian cancer, the present inventors confirmed for the first time that when porous silicon nanoparticles containing an anti-miRNA-21 oligonucleotide to which a specific cancer cell surface protein-binding peptide is conjugated are applied, apoptosis is induced in an ovarian cancer cell line and cell viability is reduced, thus, an anti-miRNA delivery system, which is the aforementioned conjugate, is expected to be usefully used as a platform for treating various cancers, especially for treating ovarian cancer.

Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

The present disclosure relates to use of an aspirin compound in facilitating exogenous nucleic acid delivery and/or expression.

Provided is an application of an amino acid as a vehicle for delivery of a nucleic acid into the nervous system. By local injection, preferably, combined with means of stereotactic injection, immunofluorescence staining, confocal microscopy, etc., the single-stranded oligonucleotides are efficiently delivered into brain cells, and the plasmid vectors are also delivered into astrocytes in mouse brain.

The present invention pertains to an inventive release mechanism for extracellular vesicle (EV)-mediated intracellular and intramembrane delivery of therapeutic polypeptides. More specifically, the invention relates to EVs comprising polypeptide constructs which comprise a therapeutic polypeptide releasably attached to an exosomal polypeptide. Furthermore, the present invention pertains to manufacturing methods, pharmaceutical compositions, medical uses and applications, and various other embodiments related to the inventive EVs.

The present invention relates to compositions and methods for treating ocular disorders. More specifically, the method includes non-surgical administration of gene therapies to the suprachoroidal space of the eye of a subject who is suffering from an ocular disorder.

Adeno-associated virus (AAV) Clade F vectors or AAV vector variants (relative to AAV9) for precise editing of the genome of a cell and methods and kits thereof are provided. Targeted genome editing using the AAV Clade F vectors or AAV vector variants provided herein occurred at frequencies that were shown to be 1,000 to 100,000 fold more efficient than has previously been reported. Also provided are methods of treating a disease or disorder in a subject by editing the genome of a cell of the subject via transducing the cell with an AAV Clade F vector or AAV vector variant as described herein and further transplanting the transduced cell into the subject to treat the disease or disorder of the subject. Also provided herein are methods of treating a disease or disorder in a subject by in vivo genome editing by directly administering the AAV Clade F vector or AAV vector variant as described herein to the subject.

The present invention relates to expression constructs and vectors for the treatment and/or prevention of diseases that are associated with a loss of NPC1 function, such as the lysosomal storage disorder Niemann-Pick type C (NPC) disease.