Patent classifications
C12N2830/60
GENE EXPRESSION SYSTEM USING STEALTHY RNA, AND GENE INTRODUCTION/EXPRESSION VECTOR INCLUDING SAID RNA
The present invention enables simultaneous and stable expression of a plurality of foreign genes by using a stealthy RNA gene expression system that is a complex that does not activate the innate immune mechanism and is formed from an RNA-dependent RNA polymerase, a single-strand RNA binding protein, and negative-sense single-strand RNAs including the following (1) to (8): (1) a target RNA sequence that codes for any protein or functional RNA; (2) an RNA sequence forming a noncoding region and derived from mRNA expressed in animal cells; (3) a transcription initiation signal sequence recognized by the RNA-dependent RNA polymerase; (4) a transcription termination signal sequence recognized by the polymerase; (5) an RNA sequence containing a replication origin recognized by the polymerase; (6) an RNA sequence that codes for the polymerase and of which codons are optimized for the species from which an introduction target cell is derived; (7) an RNA sequence that codes for a protein for regulating the activity of the polymerase and of which codons are optimized for the species from which the introduction target cell is derived; and (8) an RNA sequence that codes for the single-strand RNA binding protein and of which codons are optimized for the species from which the introduction target cell is derived.
Genetic Construct
A genetic construct comprises a promoter operably linked to a first coding sequence, which encodes tyrosine hydroxylase (TH), and a second coding sequence, which encodes GTP cyclohydrolase 1 (GCH1), wherein the second coding sequence is 3 to the first coding sequence, and the first and second coding sequences are part of a single operon. The genetic construct does not encode aromatic amino acid decarboxylase (AADC).
Turkey herpesvirus vectored recombinant containing avian influenza genes
The present invention provides a recombinant turkey herpesvirus modified by the presence of the cDNA encoding the hemagglutinin protein of avian influenza virus under a promoter. A poultry vaccine comprising the recombinant turkey herpesvirus described in the present invention can induce serological responses that may be easily detected by the hemagglutination inhibition assay but not by commercially available diagnostic ELISA kits; thus enabling easy differentiation between vaccination and field infection.
STABLE CELL LINES FOR RETROVIRAL PRODUCTION
The invention relates to retroviral producer cells comprising nucleic acid sequences encoding: gag and pol proteins; envelope protein or a functional substitute thereof; and the RNA genome of the retroviral vector particle, wherein the nucleic acid sequences are all located at a single locus within the retroviral producer cell genome.
Nucleic acid vector
The present invention provides a nucleic acid vector referred to as pVec constructed through molecular biotechnologies. pVec contains CMV enhancer/promoter, T7 promoter, 5UTR, MCS, 3UTR, poly A (120A)-TTATT, BGH poly (A) signal, kanamycin resistance gene and pUC origin, etc. So pVec can be used as a vector for both DNA vaccines or therapeutic drugs and mRNA vaccines or mRNA therapeutic drugs. The 5UTR, 3UTR and poly A (120A)-TTATT of pVec can be added to the 5 and 3 ends of the in vitro transcribed mRNA respectively and further stabilize the transcribed mRNA. The present invention also provides the constructed pVec-GM-CSF, pVec-hIL-12 and pVAX1-hIL-12, which are used for evaluating the benefits of pVec.
STABLE CELL LINES FOR RETROVIRAL PRODUCTION
The invention relates to retroviral producer cell comprising nucleic acid sequences encoding: gag and pol proteins; envelope protein or a functional substitute thereof; amplifiable selection marker; and the RNA genome of the retroviral vector particle, wherein said nucleic acid sequences are all integrated at a single locus within the retroviral producer cell genome. The invention also relates to nucleic acid vectors comprising a non-mammalian origin of replication and the ability to hold at least 25 kilobases (kb) of DNA, characterized in that said nucleic acid vector comprises retroviral nucleic acid sequences encoding: gag and pol proteins, and an env protein or a functional substitute thereof. The nucleic acid vector additionally comprises nucleic acid sequences encoding an amplifiable selection marker. The invention also relates to uses and methods using said nucleic acid vector in order to produce stable retroviral packaging and producer cell lines.
COMPOSITIONS AND METHODS FOR INHIBITING ALPHA-SYNUCLEIN AGGREGATION
Provided herein are methods and compositions useful for the treatment of neurodegenerative diseases that include adeno-associated viral vectors that encode one or more peptides that inhibit -synuclein aggregation. Such peptides are useful for treating neurodegenerative diseases such as Alzheimer's disease, a Lewy body disorder, Parkinson's disease (PD), PD with dementia (PDD), pure autonomic failure (PAF), and multiple system atrophy (MSA).
BCMA CHIMERIC ANTIGEN RECEPTORS
The invention provides improved compositions for adoptive T cell therapies for B cell related conditions.
Promoters for high level expression
The present invention provides for functional chimeric gene regulatory units capable of driving strong and sustained heterologous gene expression.
IL-12 IMMUNOTHERAPY FOR CANCER
Compositions and methods for delivering immune modulatory molecules to result in a therapeutic effect are disclosed. The compositions and methods use stably integrating lentiviral delivery systems. The methods are useful for therapeutically and prophylactically treating cancer such as leukemia.