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.

An isolated, non-naturally occurring cell-penetrating peptide (CPP) comprising the amino acid sequence:

TABLE-US-00001 [SEQ ID NO: 1] RRSRTARAGRPGRNSSRPSAPR
and sequences which have at least 60% similarity to SEQ ID NO: 1.

Isolated peptides capable of reducing the amount of dexamethasone-induced spleen and/or thymus weight loss in a mouse are disclosed. Uses thereof for treating inflammatory or degenerative diseases are also disclosed.

Described are peptides and peptide conjugates comprising CN binding motifs (CNBM) which inhibit the CN-NFAT interaction. In some embodiments, the peptides comprise: (i) CNBM; (ii) a hydrophobic, non-peptidic moiety (RH) which interacts with the hydrophobic pocket on a CN protein; (iii) a sequence -AAU1-AAU2-AAU3-AAU4-AAU5-AAU6-, wherein each of AAU2, AAU3, AAU4, AAU5, and AAU6, is, independently, optional, and each of AAU1, AAU2, AAU3, AAU4, AAU5, and AAU6 when present is independently an amino acid as defined herein; or (iv) combinations thereof. In some embodiments, RH is conjugated to the N- or C-terminus of the CNBM. In some embodiments, the sequence -AAU1-AAU2-AAU3-AAU4-AAU5-AAU6- is conjugated to the N- or C terminus of the CNBM. In some embodiments, the peptides comprise: CNBM and RH. In some embodiments. In some embodiments, the peptides comprise: CNBM and AAU1-AAU2-AAU3-AAU4-AAU5-AAU6-. In some embodiments, the peptides of the disclosure CNBM and RH.

The invention relates to a recombinant adenovirus comprising an albumin-binding moiety on the outer surface of the adenoviral hexon protein, pharmaceutical compositions containing it and its medical use. Particularly, the invention relates to an oncolytic adenovirus comprising a sequence encoding an albumin-binding moiety inserted in the hypervariable region 1 (HVR1) of the hexon protein coding sequence and its use in the prevention and/or treatment of cancer.

Compositions for CDKL5 gene therapy are provided, as well as recombinant CDKL5 proteins. Such CDKL5 gene therapy compositions and/or recombinant CDKL5 proteins may incorporate cell-penetrating polypeptides and/or leader signal polypeptides. Also provided are methods of producing such gene therapy compositions and recombinant CDKL5 proteins, as well as pharmaceutical compositions, methods of treatment, and uses of the gene therapy compositions and recombinant CDKL5 proteins.

The present invention includes a milk-derived polypeptide derivative, composition and its method for using. The milk-derived polypeptide together with its cell penetrating peptide derivatives can slow down body weight gain of mice induced by high-fat diet, reduce blood glucose, serum triglycerides and insulin levels, and improve insulin sensitivity and glucose tolerance. In addition, it can function to reduce body weight and blood glucose of already established obese mice model. Therefore, it has potentials to prepare drugs, health care products and food additives for preventing and treating obesity and its complications and other related diseases.

The present disclosure relates to a multi-functional nanoparticle targeted to breast cancer, a preparation method and use thereof. The multi-functional nanoparticle includes a targeting carrier and a medicament loaded on the targeting carrier; and the targeting carrier is made from recombinant ferritin. Cell experiments verify that the multi-functional nanoparticle has better efficacy and drug release capacity for cancer cells than those of conventional ferritin as a vector. Moreover, the drug delivery system can further achieve optical imaging of tumor cells by loading quantum dots, thus playing a role in cancer diagnosis and treatment.

Disclosed are a type of mitochondria-targeted polypeptides, the preparation method and the uses thereof. The polypeptide is abbreviated as MTP. The synthesis method of the present disclosure is simple, and the mitochondria-targeted polypeptide prepared by the method can specifically target the mitochondria of cells and are basically non-toxic to cells. In addition, these synthesized polypeptides demonstrate good cell-membrane-penetrating properties, and can conveniently undergo further multi-functional derivation and modification, thereby providing a potential delivery tool for the preparation of a mitochondria-targeted medicament.

Described herein are peptides and variants and mutants thereof capable of interacting with TEAD, disrupting the HIPPO pathway, or modulating the activity or function of TEAD interactions in a cell. Pharmaceutical compositions and uses of peptides, as well as methods of designing and manufacturing such peptides, to treat cancer, tumor, or any other disease/condition associated with a dysregulated HIPPO pathway or uncontrolled cell growth are also described herein.