A carbon nanotube composite vector having a synergistic effect of photothermal therapy and gene therapy, a preparation method therefor, and an application thereof. The vector includes a vector moiety and a gene, and the vector moiety includes carbon nanotubes, a peptide lipid, and/or an additive. A modifier is immobilized on the carbon nanotubes by a self-assembly process to prepare the composite vector that can carry and transfer the gene. The composite vector overcomes the problems that pure carbon nanotubes have poor water solubility, low biocompatibility, and poor gene carrying and transfer efficiency; moreover, the composite vector has higher photothermal conversion performances and gene transfer efficiency, reduces cytotoxicity of carbon nanotubes, and alleviates the problem of localized accumulation of carbon nanotubes. The synergistic effect of photothermal therapy and gene therapy is applied to resolve the problem in tumor treatment that the efficacy of a single treatment method is poor.

The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

A compound comprising formula (I):

##STR00001## wherein R.sup.1 is an akylamino group or a group containing at least one aromatic group; R.sup.2 and R.sup.3 are independently an aliphatic group or hydrophobic group; R.sup.4 and R.sup.5 are independently H, a substituted or unsubstituted akyl group, an akenyl group, an acyl group, an aromatic group, polymer, a targeting group, or a detectable moiety; a, b, c, and d are independently an integer from 1 to 10; and pharmaceutically acceptable salts thereof.

Disclosed are cationic lipids which are compounds of Formula I. Cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

##STR00001##

The present disclosure provides one or more amino lipids such as an amino lipids containing a sulfonic acid or sulfonic acid derivative of the formulas:

##STR00001##

wherein the variables are as defined herein. These amino lipids may be used in compositions with one or more helper lipids and a nucleic acid therapeutic agent. These compositions may be used to treat a disease or disorder such as cancer, cystic fibrosis, or other genetic diseases.

The present invention provides stable, dry powder messenger RNA formulations for therapeutic use, and methods of making and using the same.

The present invention provides, in part, a biodegradable compound of formula I, and sub-formulas thereof: Formula (I) or a pharmaceutically acceptable salt thereof, where each X independently is O or S, each Y independently is O or S, and each R.sup.1 independently is defined herein; and a liposome composition comprising the cationic lipid of formula I or a sub-formula thereof, and methods of delivering agents, such as nucleic acids including mRNA, in vivo, by administering to a subject the liposome comprising the cationic lipid of formula I or a sub-formula thereof, where the agent is encapsulated within the liposome.

##STR00001##

Provided herein are compositions and methods for delivering non-viral, capsid-free DNA vectors (ceDNA) to cytosol of a target cell in subject while reducing or inhibiting an immune response.

This disclosure relates to mRNA therapy for the treatment of arginase deficiency (AD). mRNAs for use in the invention, when administered in vivo, encode arginase 1 (ARG1). mRNA therapies of the disclosure increase and/or restore deficient levels of ARG1 expression and/or activity in subjects. mRNA therapies of the disclosure further decrease abnormal accumulation of ammonia associated with deficient ARG1 activity in subjects.

The present disclosure relates to compositions and methods for regulating the immune system and for treating cancers and other immune disorders.