The present disclosure features methods and compositions for treating Alzheimer's disease. The disclosed methods comprise administering to a subject having or suspected of having Alzheimer's a hematopoietic stem progenitor cell expressing at least one neuroprotective agent, such as ApoE2, Trem2, and/or a metallothionein.

A novel cytokine-derived cell penetrating peptide and use thereof are provided. The cell penetrating peptide has the ability to effectively deliver a biologically active substance into phagocytes, particularly macrophages, both in vitro and in vivo. The cell penetrating peptide can deliver a biologically active substance into macrophages with high efficiency compared to TAT peptide and dNP2 peptide that are commercially available as cell penetrating peptides.

The present invention relates to adeno-associated virus (AAV) vectors modified by the covalent coupling of at least one compound comprising a lactam moiety (e.g., β-lactam) to at least one amino group of an amino acid residue of the capsid of the AAV vectors. The AAV vectors are useful in transducing a cell, especially for gene therapy.

The disclosure pertains to the field of molecular means capable of binding to, and preferably of chelating, cGMP, appropriate for use in vitro or in vivo and preferably capable of targeting specific cellular compartments. The polypeptides of the disclosure comprise a chimeric construction derived from the N terminus part of PKG-Iα and PKG-Iβ, and the two cGMP binding sites of the wild type PKG.

The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.

The present invention relates to a DNA construct encoding one or more human IGF-1 isoforms that can be used for treatment of neuropathy. Further provided herein are a pharmaceutical composition including the DNA construct as an active ingredient and a method of administering the DNA construct for treatment of neuropathy. The present invention provides a safe and effective way of treating neuropathic patients.

Provided are variant adeno-associated virus (AAV) capsid proteins and recombinant AAV virions having one or more variant AAV capsid proteins. Also provided are compositions and methods for the use of the recombinant AAV virions, such as for the treatment or prophylaxis of a disease or disorder.

The present disclosure relates to methods and compositions comprising derivatized-chitosan polyplexes reversibly coated with a polyanion-containing block co-polymer for the localized expression of IL-12 in mucosal tissues, preferably in combination with an IFN-1 activator/inducer, for use in cancer immunotherapy.

Provided herein are combination therapies involving co-expression of an E2 subunit of a branched-chain alpha-keto acid dehydrogenase (BCKDH) from a skeletal muscle-targeted rAAV.hDBT vector and a liver-targeted rAAV.hDBT vector. Also provided herein are combination therapies wherein an E1a and/or an E1b subunit of the BCKDH complex is expressed from muscle and/or liver following rAAV-mediated delivery targeted to these tissues. Further provided is a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, and a method of treating a human subject diagnosed with MSUD.

Described herein are compositions comprising AAV vectors comprising a sequence encoding mucolipin 1, and methods of use thereof for gene therapy of Mucolipidosis IV (MLIV).