Biodegradable particles for delivering a nucleic acid encoding gene-editing factors or a nucleic acid associated with a therapeutic protein to a cell, and compositions, methods, systems, and kits for gene editing in vivo or ex vivo or gene therapy for treating retinal eye diseases are disclosed.

Provided herein are compositions, systems, and methods for delivering an effector protein into a cell. The present disclosure, in some aspects, provide novel proteins delivering an effector protein into a cell. The novel proteins are supernegatively charged proteins derived from highly anionic proteins identified from the proteome (e.g., human proteome). The novel protein tags can be associated (e.g., covalently or nocovalently) with the protein to be delivered to facilitate delivery of the effector protein into a cell.

Disclosed are immunogenic constructs including: a nanoparticle; a cationic polymer electrostatically bound to an exterior surface of the nanoparticle and a stabilizer bound to the cationic polymer or the exterior surface of the nanoparticle; and an antigen or antigen producing agent. Optionally, the constructs may include adjuvant and/or one or more functional oligonucleotide(s) (e.g., siRNA or pDNA). Also disclosed are methods of using the provided immunogenic constructs for co-delivering an adjuvant, antigen, and optionally siRNA to a cell, inducing immune response in a subject, and treating or preventing an infectious disease in a subject.

Primarily aimed at providing a novel pharmaceutical composition. The present invention relates to a pharmaceutical composition that contains a cerium compound as an active ingredient.

New techniques for remote administration of medicine and medical devices using an external guidance and activation system are provided. In some embodiments, medicine(s) and/or medical device(s) is/are energized to a predetermined threshold energy level by externally applied radiation, and then driven into the treatment target. The design of such device(s) (e.g., injectable machine(s)) may include sub-device(s), e.g., medical payload-carrying reservoir(s), injector(s) and abrasive tool(s), which may be activated magnetically and/or by such radiation. In some aspects, such sub-device(s) include actuable housing(s) and/or other sub-tool(s), delivering drugs to specific locations commanded by a control system or a user. In other aspects, a medicine and/or device is provided with multiple dipoles, each oriented differently in three-dimensional space, allowing a guidance control system, remote from the medicine or device, to drive the movement and three-dimensional orientation of the medical agent or particle according to a three-dimensional path.

New techniques for remote administration of medicine and medical devices using an external guidance and activation system are provided. In some embodiments, medicine(s) and/or medical device(s) is/are energized to a predetermined threshold energy level by externally applied radiation, and then driven into the treatment target. The design of such device(s) (e.g., injectable machine(s)) may include sub-device(s), e.g., medical payload-carrying reservoir(s), injector(s) and abrasive tool(s), which may be activated magnetically and/or by such radiation. In some aspects, such sub-device(s) include actuable housing(s) and/or other sub-tool(s), delivering drugs to specific locations commanded by a control system or a user. In other aspects, a medicine and/or device is provided with multiple dipoles, each oriented differently in three-dimensional space, allowing a guidance control system, remote from the medicine or device, to drive the movement and three-dimensional orientation of the medical agent or particle according to a three-dimensional path.

The present disclosure relates to antigen presenting cells and uses thereof for treating sepsis. In some aspects, disclosed herein is an antigen presenting cell, comprising: a lipid-based nanoparticle, comprising: a recombinant polynucleotide, comprising: a first nucleic acid encoding an antimicrobial peptide; a second nucleic acid encoding cathepsin B; and a third nucleic acid encoding a linker; and a vitamin-lipid.

The present application provides methods and compositions for treating cancer by administering a composition comprising nanoparticles that comprise an mTOR inhibitor (such as a limus drug) and a carrier protein (such as an albumin) based upon the mutation status of TSC1 or TSC2 and one of VHL, RBI, PBRIM1, K.DM6A, RET, SETD2 ARID 1 A, BAP1, BRCA2, TP53, RBI, ATRX, FLT1, NTRK1, TLX3, KDM6A, CDH4, CDKN2C, DAXX, ERBB3, GNAS, IL7R, PDGFRB, PMS2, PTEN, SMARCA4, and YY1AP1.

Compositions, systems and methods for delivering CRISPR/Cas9-based genome editing system and a donor protein to a cell.

Disclosed herein compositions of polysaccharides chemically cross-linked by aromatic dialdehydes. The compositions may be in form of polymeric sheets for a variety of applications. Disclosed also nano-sized particles comprising the polysaccharide chemically cross-linked by aromatic dialdehydes. The nano-sized particles may further comprise lipids and surfactants. Intranasal delivery of the nano-sized particles enables delivery of biologically active agents into the brain. Topical and transdermal delivery of the nano-sized particles enables delivery of biologically active agents for treatment of systemic or dermatological disorders. Methods of manufacturing and uses of the compositions are also disclosed.