摘要:
Skin cells are genetically altered to express a gene product encoded by an introduced polynucleotide. Specifically, the invention involves introduction of a nucleotide of interest into a skin cell by topical application of a polynucleotide that is substantially free of liposomes or charged lipids, where the skin can be either treated or untreated prior to application. Similarly, the invention involves introduction of a nucleotide of interest into a skin cell by topical application of a polynucleotide formulation comprising a liposome or charged lipid, wherein the skin is not treated by removal of hair prior to application. The method of the invention serves as a platform for delivering polynucleotides to skin cells for expression therein for any of a variety of uses including, but not limited to genetic immunization.
摘要:
The present invention is directed to the MMSC1 gene, its protein product and the use of the protein to (i) detect mutant MMAC1 proteins, (ii) screen for drugs which can be used for suppressing tumor growth and (iii) identify proteins which interact with the MMAC1 gene or are involved in the tumor suppression pathway of the MMAC1 gene.
摘要:
The present invention provides compositions and methods for utilizing recombinant alphavirus particles. In particular, it provides a recombinant alphavirus particle which is free from contaminating wild-type alphavirus particles.
摘要:
A two-step, scalable method is described for identifying the cellular function(s) of one or more genes of unknown function, and for identifying modulators or the gene(s). The method uses the reversal or alteration of a phenotype created by the expression of the heterologous gene, e.g., a human gene, to identify modulators of that gene's activity. That modulator is then used in an in vitro or in vivo disease model system to identify compounds which affect disease progression. The subset of compounds which influence disease in a therapeutic manner are drug leads. However, all compounds which influence disease progression are tools to at least partially characterize gene function. The inhibitor identification step or the method is preferably carried out using a plurality of microbial strains or cell lines expressing different heterologous DNA sequences in a single solution. The method is particularly useful for genes which have not been validated as good inhibitor targets.
摘要:
The invention provides methods of evolving nucleic acids for use in gene therapy by recursive sequence recombination. Many of the methods evolve vectors, both viral and nonviral, to have improved properties. For example, vectors are evolved to have improved properties of viral titer, infectivity, expression of a gene within a vector, tissue specificity, viral genome capacity, episomal retention, lack of immunogenicity of the vectors or an expression product thereof, site-specific integration, increased stability, or capacity to confer cellular resistance to microorganism infection. The invention further provides an isolated O6-methylguanine-DNA methyltransferase (MGMT) enzyme.
摘要:
Secreted proteins can be identified using a method which exploits the ability of microsomes to modify proteins post-translationally. Nineteen human secreted proteins and full-length cDNA sequences encoding the proteins have been identified using this method. The proteins and cDNA sequences can be used, inter alia, for targeting other proteins to the membrane or extracellular milieu.
摘要:
The present invention pertains generally to novel Neisseria meningitidis serogroup B glycoconjugates. More particularly, the invention pertains to glycoconjugates formed from a Neisseria meningitidis serogroup B capsular oligosaccharide derivative (MenB OS derivative) in which sialic acid residue N-acetyl groups are replaced with N-acyl groups. The invention also pertains to vaccine formulations containing the glycoconjugates, methods of making the vaccine formulations, and methods of using the vaccine formulations to treat or prevent Neisseria meningitidis serogroup B or E. coli K1 disease in a mammalian subject.
摘要:
Provided are methods for selectively expressing therapeutic molecules, such as secretory proteins, antisense molecules and ribozymes, in the liver. The methods find use in treating hepatic diseases or conditions. The methods also find use in treating any disease or condition in which systemic administration of the therapeutic substance, for example, a secretory protein, is desired. The methods involve administering to a mammalian patient having a need for liver expression of a therapeutic molecule an AAV vector containing a therapeutically effective amount of the therapeutic molecule. Also provided are novel vectors employable in these methods.