Variagenics, Inc.

Nuvelo, Inc.

Sequenom, Inc.

Other

Thiogenesis Therapeutics, Inc.

Antolrx, Inc.

Vincent P Stanton, Jr

Gregory Lawrence Verdine

Jia Liu Wolfe

Tomohiko Kawate

Jeffrey Charles Olson

James P Basilion

Charles Allerson

Martin Zillmann

Patrice P Rioux

Ling X Shen

Brett P Monia

David E Housman

Fred David Ledley

Piotr Barski

Dariusz Witt

Aditi Jhaveri

David Steffen

Robert Mark Adams

Martin Zillman

The invention provides compositions (e.g., liposomes) for treating diseases and conditions associated with pathologic immune responses and methods for formulating and administering such compositions.

Tolerogenic liposomes and methods of use thereof

The invention features compositions, methods, and kits containing (i) one or more cysteamine precursor compounds convertible to cysteamine in vivo, and (ii) optionally agents to enhance that conversion, formulated to produce a spectrum of pharmacokinetic profiles of cysteamine that can be tailored to individual patients and diseases. The invention also features varying modes of administration of the therapeutic substances in the treatment of cystinosis and other cysteamine sensitive disorders. In particular, formulations combining active ingredient(s) with pharmaceutical excipients that permit sustained cysteamine plasma concentrations are featured.

Compositions for controlled release of cysteamine and systemic treatment of cysteamine sensitive disorders

The invention features methods for the treatment of cystinosis and other cysteamine sensitive disorders in a subject including administration of a disulfide convertible to cysteamine in vivo. The methods can include the separate administration of a reducing agent to the subject to increase the bioavailablity and extend the plasma pharmacokinetic profile of the cysteamine produced following administration of the disulfide. The methods permit sustained cysteamine plasma concentrations in a subject.

Methods for the treatment of cysteamine sensitive disorders

Described are methods for identifying targets in RNA molecules based on differences in RNA secondary structure related to sequence variances between different allelic forms. Also described are methods for identifying or developing small molecules which can be used to modulate a target RNA by creation of protein-small-molecule-RNA complexes, as well as compositions which include such small molecules and methods of using those small molecules and compositions.

Methods for targeting RNA molecules

Methods for determining genotypes and haplotypes of genes are described. Also described are single nucleotide polymorphisms and haplotypes in the ApoE gene and methods of using that information.

Methods for genetic analysis of DNA to detect sequence variances

The presently claimed invention concerns a method for biasing the amplification of the DNA molecules in a sample such that a nucleic acid molecule having a specific nucleotide at a selected position (e.g., a polymorphic site) is preferentially amplified relative to an otherwise identical nucleic acid molecule not having the specific nucleotide at the selected position. The method entails amplification of target nucleic acid molecules using a pair of primers one of which causes the incorporation into the amplification product of a sequence that interferes with amplification of nucleic acid molecules not having the specific nucleotide at the selected position. The method is useful for preferentially amplifying at least a portion of one allele of a gene relative to another, different allele of the gene in a sample containing both alleles of the gene.

Restriction enzyme genotyping

The present invention relates to methods for the detection of polymorphisms by substituting a base-modified nucleotide for a natural nucleotide in a polynucleotide. The base-modified nucleotide renders the polynucleotide more susceptible to cleavage at the sites of its incorporation than site consisting of natural nucleotides. The fragments obtained are then analyzed to determine the presence or absence of a polymorphism.

Base-modified nucleotides and their use for polymorphism detection

The present invention relates to a method for genotyping a diploid organism to detect single nucleotide polymorphisms (SNPs) using modified nucleotides or nucleotide residues substituted with fluorescent groups.

Fluorescence-based genotyping

The present invention relates to a method for genotyping a diploid organism by cleaving segments of two alleles such that 7-20 nucleotide fragments that contain a suspected polymorphic locus are produced and comparing the masses of those fragments.

Genotyping by mass spectrometric analysis of allelic fragments

The present disclosure describes the use of genetic variance information for folate transport or metabolism genes or pyrimidine transport or metabolism genes in the selection of effective methods of treatment of a disease or condition. The variance information is indicative of the expected response of a patient to a method of treatment. Methods of determining relevant variance information and additional methods of using such variance information are also described.

Thymidine phosphorylase gene sequence variances having utility in determining the treatment of disease

Methods are described for identifying an RNA target for an allele-specific RNA inhibitor by identifying a sequence variance in an RNA encoded by a gene of interest, and determining whether the secondary structure of the RNA comprising the variance differs from that of an otherwise identical RNA not comprising the variance.

Disclosed are methods and reagents for detecting nucleotide mismatches (for example, due to sequence variances) in a nucleic acid sample involving the use of a nucleic acid probe derived from a hemizygous cell. Methods for determining the haplotype of a nucleic acid sample are also disclosed. Also disclosed are methods for producing the probe and kits containing the probe.

Growing community of inventors

Belmont, MA, United States of America

Vincent P Stanton, Jr