Patent application number | Description | Published |
20080269149 | Chimeric Vectors - The present invention is based, in part, on the discovery that parvovirus (including AAV) capsids can be engineered to incorporate small, selective regions from other parvoviruses that confer desirable properties. The inventors have discovered that in some cases as little as a single amino acid insertion or substitution from a first parvovirus (e.g., an AAV) into the capsid structure of another parvovirus (e.g., an AAV) to create a chimeric parvovirus is sufficient to confer one or more of the desirable properties of the first parvovirus to the resulting chimeric parvovirus and/or to confer a property that is not exhibited by the first parvovirus or is present to a lesser extent. | 10-30-2008 |
20090004743 | HELPER VIRUS-FREE AAV PRODUCTION - A method for the production of adeno-associated virus stocks and recombinant adeno-associated virus stocks that are substantially free of contaminating helper virus is described. The method utilizes transfection with helper virus vectors to replace the infection with helper virus used in the conventional method. | 01-01-2009 |
20090104155 | TREATMENT OF CONNECTIVE TISSUE DISORDERS - The present invention relates to methods of delivering nucleic acids to connective tissue cells and to methods of treating connective tissue disorders; in particular, the invention provides methods of delivering nucleic acids to connective tissue cells and methods of treating connective tissue disorders using parvovirus vectors. | 04-23-2009 |
20090215879 | METHODS AND COMPOSITIONS FOR ADENO-ASSOCIATED VIRUS (AAV) WITH HI LOOP MUTATIONS - The invention provides modified AAV capsid proteins comprising substitutions in the HI loop. Suitable substitutions include affinity tags, sequences that facilitate detection and/or targeting peptides. The invention also provides virus capsids and virus vectors comprising the modified AAV capsid proteins and methods of using the same. Further provided are methods of purifying the modified AAV capsid subunits, virus capsids and virus vectors of the invention. | 08-27-2009 |
20100137211 | METHODS AND COMPOSITIONS FOR INTRA-ARTICULAR COAGULATION PROTEINS - The present invention provides methods and compositions for treating blood clotting factor disorders and/or reducing bleeding-associated joint damage by treatments delivered to the joint in a subject. | 06-03-2010 |
20100303776 | Methods and compositions for regulated expression of multiple nucleic acids - The present invention provides a cell comprising a first heterologous nucleic acid construct and a second heterologous nucleic acid construct, wherein each of said first and second heterologous nucleic acid constructs comprises: A. a first nucleotide sequence encoding a nucleotide sequence of interest (NOI); and B. a second intronic nucleotide sequence comprising: i) a first set of splice elements defining a first intron that is removed by splicing to produce a first RNA molecule encoded by said first nucleotide sequence in the absence of activity at a second set of splice elements; and ii) the second set of splice elements defining a second intron different from said first intron, wherein said second intron is removed by splicing to produce no RNA molecule and/or to produce a second RNA molecule that is not encoded by said first nucleotide sequence, wherein each said first nucleotide sequence of each of said first and second heterologous nucleic acid constructs is different from one another and wherein each said second intronic nucleotide sequence of each of said first and second heterologous nucleic acid constructs is different from one another. | 12-02-2010 |
20100310516 | Duplexed Parvovirus Vectors - The present invention provides duplexed parvovirus vector genomes that are capable under appropriate conditions of forming a double-stranded molecule by intrastrand base-pairing. Also provided are duplexed parvovirus particles comprising the vector genome. Further disclosed are templates and methods for producing the duplexed vector genomes and duplexed parvovirus particles of the invention. Methods of administering these reagents to a cell or subject are also described. Preferably, the parvovirus capsid is an AAV capsid. It is further preferred that the vector genome comprises AAV terminal repeat sequences. | 12-09-2010 |
20110104119 | Chimeric Vectors - The present invention is based, in part, on the discovery that parvovirus (including AAV) capsids can be engineered to incorporate small, selective regions from other parvoviruses that confer desirable properties. The inventors have discovered that in some cases as little as a single amino acid insertion or substitution from a first parvovirus (e.g., an AAV) into the capsid structure of another parvovirus (e.g., an AAV) to create a chimeric parvovirus is sufficient to confer one or more of the desirable properties of the first parvovirus to the resulting chimeric parvovirus and/or to confer a property that is not exhibited by the first parvovirus or is present to a lesser extent. | 05-05-2011 |
20120009268 | Modified Virus Vectors and Methods of Making and Using the Same - The present invention provides AAV capsid proteins (VP1, VP2 and/or VP3) comprising a modification in the amino acid sequence in the three-fold axis loop 4 and virus capsids and virus vectors comprising the modified AAV capsid protein. In particular embodiments, the modification comprises a substitution of one or more amino acids at amino acid positions 585 to 590 (inclusive) of the native AAV2 capsid protein sequence or the corresponding positions of other AAV capsid proteins. The invention also provides methods of administering the virus vectors and virus capsids of the invention to a cell or to a subject in vivo. | 01-12-2012 |
20130012574 | Compositions and Methods for Enhanced Parvovirus Transduction - The present invention provides methods and compositions for enhanced transduction of an adeno-associated virus (AAV) vector comprising a heterologous nucleic acid of interest wherein the AAV vector genome is oversized relative to a wild type AAV genome by employing a proteasome inhibitor. | 01-10-2013 |
20130109742 | Restrictive Inverted Terminal Repeats for Viral Vectors | 05-02-2013 |
20130252325 | Duplexed Parvovirus Vectors - The present invention provides duplexed parvovirus vector genomes that are capable under appropriate conditions of forming a double-stranded molecule by intrastrand base-pairing. Also provided are duplexed parvovirus particles comprising the vector genome. Further disclosed are templates and methods for producing the duplexed vector genomes and duplexed parvovirus particles of the invention. Methods of administering these reagents to a cell or subject are also described. Preferably, the parvovirus capsid is an AAV capsid. It is further preferred that the vector genome comprises AAV terminal repeat sequences. | 09-26-2013 |
20140242671 | Cell Line for Production of Adeno-associated Virus - This invention relates to a HEK293 cell line that grows under animal component-free suspension conditions. The cell line is ideal for rapid and scalable production of adeno-associated virus (AAV) and supports production of all serotypes and chimera of AAV. | 08-28-2014 |
20140271551 | Synthetic Adeno-Associated Virus Inverted Terminal Repeats - This invention relates to synthetic adeno-associated virus (AAV) inverted terminal repeats (ITRs) that exhibit altered activities compared to a naturally occurring AAV ITR and methods of using the same for delivery of nucleic acids to a cell or a subject. The synthetic ITRs provide a larger packaging capacity and the ability to manipulate activities such as transduction efficiency, cellular response to transduction, and transcription. | 09-18-2014 |