M2 PHARMA-July 24, 2019-Iveric bio Advances LCA10 Minigene
Programme and Expansion of Orphan Gene Therapy Portfolio with USH2A-Related Inherited Retinal Diseases Programme
A similar approach has been tested by Fest and coworkers, who have tested a survivin minigene
DNA vaccine administered using SL7207 as DNA carrier.
performed a targeted knockout of a patient-derived mutant RHO P23H minigene
in a transgenic mouse model.
Three four-day-old DGR dogs submitted to intravenous injection with an adenovirus-associated vector (AAV9) carrying a human codon with dystrophin minigene
under the control of the cytomegalovirus (CMV), presented generalized muscle transgene expression after 16 weeks of treatment (KORNEGAY et al., 2010).
(48) For in vitro functional evaluation of the exon skipping caused by the lower splicing efficiency of these variants, a minigene
assay can be used.
To investigate the involvement of G[alpha]12/13, we transiently transfected HLMVECs with the minigene
vectors (Cue Biotech, Chicago, IL) encoding a unique peptide that specifically blocks the receptor/G protein interface.
A miR-214 minigene
was produced by PCR with primers (5'-GCGCGGATCCTTTTCTCCCTT-TCCCCTTACTCT-3' and 5'-CCGGAATTCCGAGCCCCTCATTTTGGTTGTAG-3) and inserted into the pSIF-neo-IRES-GFP plasmid (provided by Dr.
The primers for SMN1-S, SMN2-S minigene
, and conserved splice site constructs are as follows: forward: SMN.Fwd (5'-ATA ATT CCC CCA CCA CCT CC-3'), reverse: pCDNA.Rev (5'-CTA GAA GGC ACA GTC GAG GCT-3') which specifically anneal to the plasmid backbone.
The SOD2-knockout allele was screened by targeting amplification of the hprt minigene
used to generate the knockout by using HPRTxl-2F (5'-CAGCCCTGGCGTCGTGAT TAGT-3') and HPRTx7R (5'-CGTGGGGTCCTTTTCACCAGCAA-3'), yielding a 2.3-kb product, and was further verified by the MSOD2x3R (5'-GAGCGACCTGAGTTGTAACATCTCC-3') and HPRTx7F (5'-GACCCCACGAAGTGTTGGATATAAG-3') primers (2.1-kb product).
The use of gene therapy is to inject a smaller yet functional version of the gene - called a minigene
- within the viral carrier.
Currently, JWCI's Experimental Therapeutics Laboratory is designing an antisense "minigene
" version of GCS.
A Multivalent minigene
vaccine, containing B-cell, cytotoxic T-lymphocyte, and Th epitopes from several microbes, induces appropriate responses in vivo and confers protection against more than one pathogen.