The present disclosure relates to compounds and methods which may be useful for modulating the expression of a target gene comprising a CGG trinucleotide repeat sequence and treating diseases and conditions in which the target gene plays an active role. The present disclosure provides compounds and methods for modulating the expression of fmr1 and fmr2, and provides compounds and methods for treating fragile X syndrome and fragile XE syndrome.

Disclosed herein are recombinant AAV variant (e.g., variant serotype 3B (AAV3B)) capsid proteins and variant capsid protein-containing viral particles with enhanced ability to transduce hepatic cells. Viral particles containing these capsid variants are capable of evading neutralization by the host humoral immune response. The recombinant AAV3B variant proteins and viral particles disclosed herein were identified from a variant AAV3B capsid library that was engineered by making substitutions in only the variable regions of the capsid. Some embodiments of the AAV3B capsid variants disclosed herein comprise the AAV3B-G3 variant and the AAV3B-E12 variant. Compositions of these variant AAV particles are provided that are useful for transducing and delivering therapeutic transgenes to cells, such as liver cells, and thus treat diseases and disorders pertaining to these cells.

Disclosed herein are recombinant AAV variant (e.g., variant serotype 3B (AAV3B)) capsid proteins and variant capsid protein-containing viral particles with enhanced ability to transduce hepatic cells. Viral particles containing these capsid variants are capable of evading neutralization by the host humoral immune response. The recombinant AAV3B variant proteins and viral particles disclosed herein were identified from a variant AAV3B capsid library that was engineered by making substitutions in only the variable regions of the capsid. Some embodiments of the AAV3B capsid variants disclosed herein comprise the AAV3B-G3 variant and the AAV3B-E12 variant. Compositions of these variant AAV particles are provided that are useful for transducing and delivering therapeutic transgenes to cells, such as liver cells, and thus treat diseases and disorders pertaining to these cells.

In some embodiments, the invention relates to methods and reagents for the identification of compounds that traverse he cell membrane of an animal cell. In some embodiments, the invention provides additional methods for determining if a candidate compound that traverses an animal cell membrane is able to modulate an intracellular target, as well as reagents and kits for reagents and kits for performing the disclosed methods.

In some embodiments, the invention relates to methods and reagents for the identification of compounds that traverse he cell membrane of an animal cell. In some embodiments, the invention provides additional methods for determining if a candidate compound that traverses an animal cell membrane is able to modulate an intracellular target, as well as reagents and kits for reagents and kits for performing the disclosed methods.

Alpha-sheet polypeptide multimers, and polypeptides for making multimers, compositions and medical devices including them, and their use for treating and diagnosing amyloid diseases or amyloid-associated diseases are disclosed.

Alpha-sheet polypeptide multimers, and polypeptides for making multimers, compositions and medical devices including them, and their use for treating and diagnosing amyloid diseases or amyloid-associated diseases are disclosed.

Provided are a surfactant adhesive protein comprising an amphiphilic peptide, as a surfactant adhesive protein, at the carbon or amine terminal, a silicone oil and an anticancer composition comprising the surfactant adhesive, where the surfactant adhesive enables homogeneous dispersion of hydrophilic or hydrophobic particles in a hydrophobic or hydrophilic solvent on the basis of strong adhesive strength of the mussel adhesive protein, and the surface adhesive can be favorably used as a surface coating agent requiring antibacterial or antiviral functions as well as a cosmetic product or an ink.

Provided are a surfactant adhesive protein comprising an amphiphilic peptide, as a surfactant adhesive protein, at the carbon or amine terminal, a silicone oil and an anticancer composition comprising the surfactant adhesive, where the surfactant adhesive enables homogeneous dispersion of hydrophilic or hydrophobic particles in a hydrophobic or hydrophilic solvent on the basis of strong adhesive strength of the mussel adhesive protein, and the surface adhesive can be favorably used as a surface coating agent requiring antibacterial or antiviral functions as well as a cosmetic product or an ink.

The present invention relates to peptides, proteins, nucleic acids and cells for use in immunotherapeutic methods. In particular, the present invention relates to the immunotherapy of cancer. The present invention furthermore relates to tumor-associated T-cell peptide epitopes, alone or in combination with other tumor-associated peptides that can for example serve as active pharmaceutical ingredients of vaccine compositions that stimulate anti-tumor immune responses, or to stimulate T cells ex vivo and transfer into patients. Peptides bound to molecules of the major histocompatibility complex (MHC), or peptides as such, can also be targets of antibodies, soluble T-cell receptors, and other binding molecules.