134results about How to "Efficient transfection" patented technology

Disclosed are improved VP2-modified recombinant adeno-associated viral (rAAV) vectors, expression systems, and rAAV virions that are fully virulent, yet lack functional VP2 protein expression. Also disclosed are pharmaceutical compositions, virus particles, host cells, and pharmaceutical formulations that comprise these modified vectors useful in the expression of therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in the cells and tissues of selected mammals, including, for example, human tissues and host cells.

The invention provides the construction for a farnesyl pyrophosphoric acid synthetase RNA (Ribonucleic Acid) interference recombinant lentivirus vector, which comprises the following steps of: sieving the most effective target sequence of an FDS (farnesyl diphosphate synthase) gene RNAi (RNA interference) in a tool cell 293T cell, synthesizing the double-stranded DNA of the most effective target sequence, connecting to a pGCSIL-GFP vector and successfully constructing the recombinant vector through enzyme cutting, sequencing and identification. Researches indicate that the constructed RNA interference vector LV-sh-FDS can downwards modulate the expression of an FDS mRNA (Messenger RNA) level in a neonatal rat cardiac myocyte, simultaneously can downwards modulate the expression of myocardial hypertrophy markers such as cell areas and marker genes beta-MHC (Myosin Heavy Chain) and BNP (Brain Natriuretic Peptide), additionally can effectively inhabit the activity of RhoA while downwards modulating the FDS, can be applied in preparing medicaments for treating myocardial hypertrophy diseases and also can be applied in preparing medicaments for cholesterol metabolic control.

Provided is a method for production of nanocrystalline hydroxyapatite particles, and nanocrystalline hydroxyapatite particles produced according to the method. The nanocrystalline hydroxyapatite particles exhibit substantially superior cell transformation abilities as compared to known and commercially-available calcium phosphate kits. The nanocrystalline hydroxyapatite particles also find use in tissue engineering applications, for example bone and tooth engineering and repair applications.

Provided herein are methods and compositions related to viral vectors. Also provided herein are methods and compositions for the efficient transfection of a host, for example through the highly efficient lentivector delivery system, and for the exquisite control of the timing and level of expression of the transferred sequence of interest by the simple administration of a modulator to the host harboring the transferred sequence of interest. Also disclosed are methods of making transgenic mice and transgenic mice made using compositions and methods relating to viral vectors.

The present invention is directed compounds for reversibly modification of biologically active molecules. Described are polyconjugates systems that incorporate targeting, anti-opsonization, anti-aggregation, and transfection activities into small biocompatible in vivo delivery conjugates. The use of reversible modification provides for physiologically responsive activity modulation.

A nanostructured molecular delivery vehicle comprising magnetic materials and configured to receive passenger biomolecules. The application of a an appropriate magnetic field having a gradient orients and drives the vehicle into a biological target, which may comprise cells, cell masses, tissue slices, tissues, etc. Under the control of the magnetic field, these vehicles can penetrate cell membranes. Then, the biomolecules carried by the vehicle can be released into the cells to perform their functions. Using this “nanospearing” technique, unprecendented high transfection efficiency has been achieved in several difficult-to-transfect cells. These include, but are not limited to, Bal 17 cells, ex vivo B cells, primary cultured cortical neurons, etc. This method advances the state of the art, providing an improved technique for the introduction of exogenous molecules to cells, with the clinical applications including, but not being limited to, drug delivery, gene therapy, vaccination, etc.

Polymers including poly(amine-co-ester), poly(amine-co-amide), or a combination thereof, and nanoparticles, particularly solid core nanoparticles, formed therefrom are provided. Solid core nanoparticles fabricated from hydrophobic polymers often require the presence of cationic complexing agents to stabilize negatively charged active agents such as siRNA. However, complexing agents are optional in the disclosed formulations because the nanoparticles contain cationic amines to stabilize negatively charged nucleic acids and hydrophobic domains to condense the nucleic acid into the core of the formed nanoparticles, thus improving encapsulation efficiency. This increase in nucleic acid loading allows the disclosed solid core nanoparticles to deliver more nucleic acid per cell without increasing total polymer delivered, further reducing cytotoxicity. Pharmaceutical compositions including an effective amount of the nanoparticles are also provided, and be used, for example, for in vitro and in vivo delivery of nucleic acids.

The invention discloses a positive ion nanostructure lipid carrier, a manufacturing method and the application of the positive ion nanostructure lipid carrier. A positive ion nanostructure lipid carrier compound has a structure in a general formula VI, wherein, 3A and A4 are hydrophilic head parts, 3A or A4 is a hydrophilic molecule formed by one or multiple same or different amino acids, and 3A and A4 are the same or different; R is a linking arm between the head part and a tail part and is of a chain-shaped or branched-chain structure; and 3B and B4 are hydrophobic tail parts, 3B or B4 is a chain-shaped hydrophobic molecule, and 3B and B4 are the same or different. The positive ion nanostructure lipid carrier compound disclosed by the invention has the characteristics of simple structure, convenient composition and the like; the positive ion nanostructure lipid carrier compound also has the characteristics of low toxicity and high transfection efficiency, the complicated process in composition is avoided, auxiliary lipidosome in other types is not needed to be used in a transfection process, the operation and the use are easy, thus the positive ion nanostructure lipid carrier compound disclosed by the invention has a potential development prospect of commercial transfection reagent.

The invention relates to a functionalized arborescent macro radical carrier system for targeting malignant cerebroma. The functionalized arborescent macro radical carrier system is obtained by taking one or more of the following combinations as the targeting functional factor: transferrin and transferrin monoclonal antibody, or polyclonal antibody, folic acid (FA) and epidermal growth factor receptor monoclonal antibody, or polyclonal antibody and RGD cyclic peptide, chemically connecting the combinations to arborescent macro radical polyamide-amine (PAMAM) carrier system, and then compounding the combinations with treatment factor oligonucleotide, siRNA molecule or plasmid DNA. Through intra-tumor stereotactic injection, carotid injection or tail vein, the distribution of genes in the malignant cerebroma can be improved, high expression can be realized, and the effective cerebroma inhibition can be realized. Compared with the commercialized gene carrier Oligofectamin and unmodified PAMAM, the FA-PAMAM can remarkably improve the targeting delivery ability of the in vitro and in vivo genes.

The invention discloses a method for preparing cells carrying exogenous molecules in a photoinduced perforating mode, a base material for preparing the cells and the cells, belongs to the field of medical instrument modification and the field of nanometer materials, and particularly relates to a reagent kit for modifying the surface of a cell culture base material (including a cell culture plate and other base materials capable of being used for culturing cells) with gold nano-materials (including gold nano particle aggregation, gold nano rods, gold nano cones and the like). The purpose of carrying out exogenous macromolecular transfer on cells cultured on the reagent kit is achieved through near-infrared band laser assistance. According to the system, gold nano particles are used for absorbing a large amount of laser energy at the near-infrared band so as to improve the membrane permeability of the cells cultured on the system; the activity of the cells can be well maintained by optimizing the laser power and the irradiation time; the reagent kit has the advantages of being simple, direct, fast and efficient, and can be applied to different cell lines for transfer of exogenous macromolecules of different types.