In various embodiments a polyrotaxane carrier for in vivo delivery of a nucleic acid is provided. In certain embodiments the carrier comprises: a multi-arm polyethylene glycol (PEG) backbone comprising at least three arms; at least one cyclic compound having a cavity, where an arm of said multi-arm PEG backbone is threaded into the cavity of said cyclic compound forming an inclusion complex; a bulky moiety capping the terminal of the arm(s) threaded into said cyclic compound where said moiety inhibits dethreading of the cyclodextrin from the arm(s) of said backbone; and where at least one arm of said PEG backbone is free of cyclic compounds; and where said carrier has a net positive charge.

The present invention realtes to lentiviral particles which have been pseudotyped with Nipah virus (NiV) fusion (F) and attachment (G) glycoproteins (NiVpp-F/G). Additionally, the present invention relates to truncated NiV-F glyocproteins useful in producing such NiVpp lentiviral particles, as well as to additional variant peptides which enhance activity. Further, the present invention relates to methods of using such lentiviral particles or sequences, for example in the treatetment of cancer or CNS disorders.

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

Compositions having nucleic acid sequences for regulating expression from gene therapy vectors, and methods of using the compositions, are provided.

Disclosed herein, in certain embodiments, are recombinant Arc and endogenous Gag polypeptides, and methods of using recombinant Arc and endogenous Gag polypeptides.

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

Described herein are CRISPR/Cas9 constructs designed for the C-terminal truncation of human amyloid precursor protein (APP) as well as methods of making and using such a construct.

The disclosure relates to novel lipidic compounds, lipid nanoparticles (LNPs) containing thereof, and the use of the lipidic compounds or the LNPs for the delivery of nucleic acid. The lipidic compounds as disclosed herein comprise at least one terminal radical of formula (I): *—NH—CX—(NH)n-A (I) wherein: —*- represents a single bond linking said radical of formula (I), directly or not, to to one C.sub.10 to C.sub.55 lipophilic or hydrophobic tail-group; —n is 0 or 1; —X is an oxygen or sulfur atom, and —A represents an optionally substituted 5- or 6-membered unsaturated heterocyclic radical or 5- or 6-membered heteroaromatic ring radical, both containing at least one nitrogen atom; or one of the pharmaceutically acceptable salts of said radical of formula (I); and with said compound that is in all the possible racemic, enantiomeric and diastereoisomeric isomer forms.

The present invention provides modified viral capsid and viral particles that contain an inserted or conjugated IR-binding agent (e.g., an IR-binding peptide). The invention also provides related polynucleotide sequences that encode such modified capsid proteins, as well as vectors for expressing the modified capsid proteins. Also provided in the invention are recombinant viral vectors or viral particles (e.g., rAAVs) having (1) a modified capsid (for non-enveloped viruses) or modified viral envelope (for enveloped viruses) that contains an inserted or conjugated IR-binding agent, and (2) a recombinant or engineered viral genome (e.g., AAV genome) that harbors a heterologous target gene or transgene sequence (e.g., a therapeutic protein encoding sequence). Further provided in the invention are methods for constructing the engineered viral vectors, and methods of using the vectors for delivering a transgene.

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.