摘要:
Methods and compositions for reducing expression of genes on Chromosome 21 (“Chr 21”) by targeting an XIST transgene to the Dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) gene or a Regulator of calcineurin 1 (RCAN1) gene, and cells and transgenic animals comprising an XIST transgene inserted into a DYRK1A or RCAN1 allele, e.g., cells and animals trisomic for human Chr 21 and mouse Chr 16.
摘要:
The subject invention provides materials and methods useful in safely and effectively preventing pathological proliferation of blood vessels. The prevention of the over-proliferation of blood vessels according to the subject invention is particularly advantageous for treatment of certain ocular conditions including age-related macular degeneration (AMD), retinopathy of prematurity (ROP) and diabetic retinopathy. In preferred embodiments, the subject invention provides materials and methods for effective treatment of pathological ocular neovascularization using gene therapy. In a specific embodiment the materials and methods of the subject invention can be used to treat AMD.
摘要:
According to some embodiments herein, expression systems and methods for activity-dependent transcription of nucleic acids are provided. In some embodiments, adeno-associated viral vector systems comprise an immediate early gene promoter operably linked to a transcriptional activator. The transcription activator can be fused to an N-terminal portion of an immediate early gene, for example fos.
摘要:
The subject invention provides materials and methods useful in safely and effectively preventing pathological proliferation of blood vessels. The prevention of the over-proliferation of blood vessels according to the subject invention is particularly advantageous for treatment of certain ocular conditions including age-related macular degeneration (AMD), retinopathy of prematurity (ROP) and diabetic retinopathy. In preferred embodiments, the subject invention provides materials and methods for effective treatment of pathological ocular neovascularization using gene therapy. In a specific embodiment the materials and methods of the subject invention can be used to treat AMD.
摘要:
The subject invention provides materials and methods useful in safely and effectively preventing pathological proliferation of blood vessels. The prevention of the over-proliferation of blood vessels according to the subject invention is particularly advantageous for treatment of certain ocular conditions including age-related macular degeneration (AMD), retinopathy of prematurity (ROP) and diabetic retinopathy. In preferred embodiments, the subject invention provides materials and methods for effective treatment of pathological ocular neovascularization using gene therapy. In a specific embodiment the materials and methods of the subject invention can be used to treat AMD.
摘要:
The present invention is directed to methods and compositions useful in producing cells and animals having a disruption or modification of a target gene. Vectors useful in producing these cells and animals are described. In addition, methods of screening and enriching cells comprising a targeted gene modification are provided.
摘要:
The present invention provides porcine adenovirus sequence essential for encapsidation and provides adenovirus vectors comprising such sequences. The present invention provides host cells and composition comprising adenovirus vectors comprising porcine adenovirus sequence essential for encapsidation as well as methods for making and using such adenovirus vectors. The present invention discloses porcine adenovirus sequence for E1 transcriptional control and provides porcine adenovirus vectors comprising a modification(s) in the E1 transcriptional control region.
摘要:
The invention generally relates to cell-specific expression vectors. It particularly relates to targeted gene therapy using recombinant expression vectors and particularly adenovirus vectors. The invention specifically relates to replication-conditional expression vectors and methods for using them. Such vectors are able to selectively replicate in a target cell or tissue to provide a therapeutic benefit in a tissue from the presence of the vector per se or from one or more heterologous gene products expressed from the vector and distributed throughout the tissue. In such vectors, a gene essential for replication is placed under the control of a heterologous tissue-specific transcriptional regulatory sequence. Thus, replication is conditioned on the presence of a factor(s) that induces transcription or the absence of a factor(s) that inhibits transcription of the gene by means of the transcriptional regulatory sequence with this vector; therefore, a target tissue can be selectively treated.
摘要:
The present invention is directed to methods and compositions useful in producing cells and animals having a disruption or modification of a target gene. Vectors useful in producing these cells and animals are described. In addition, methods of screening and enriching cells comprising a targeted gene modification are provided.
摘要:
The invention relates to a recombinantvector for the cloning and/or expression and/or transfer of an exogenous nucleotide sequence characterized in that it consists of any sequence contained in the ClaI-PvuII fragment comprising nucleotides 7702 to 1527 of the sequence given in FIG. 1 and comprising the LTR sequence included between nucleotides 7842 and 144, the PBS site starting at nucleotides 145, the packaging sequence included in the sequences of 250 nucleotides following the end of the LTR sequence, the said sequence being capable of controlling the cloning and/or expression and/or transfer at the exogenous sequence whatever its transcriptional orientation with respect to the transcriptional orientation of the virus. It relates to the use of this vector for the transfer and/or cloning and/or expression of genes, in particular in the context of gene therapy.