PLEKHM1 (cDNA) # 1 GCAAAGUCCUGGCAUCCUA # 2 GAACGCGUCUCUGUACGAG VPS11 (cDNA) GAAACGAGAUCUCCAUGAU VPS41 (cDNA) GAGAAUGAAUGUAGAGAU RAB7A (cDNA) # 1 GUACAAAGCCACAAUAGGA # 2 AAACGGAGGUGGAGCUGUA
Gln tRNA Promoter
GGTTCCATGGTGTAATGGTTAGCACTCTGGACTCTGAATCCAGCGATCCGAGTTCAAATCTCGGTGGAACCT
AAV-TBG-saCas9-U6-sgRNA Ready to Package
AAV-TBG-saCas9-U6-sgRNA Ready to Package
AAV-tRNA-sgRNA-CMV-hAsCpf1 Ready to Package
AAV-tRNA-sgRNA-CMV-hAsCpf1 Ready to Package
Several Validated siRNAs
Mouse Rab9-siRNA sense, CGGCGACUAUCCUUACUUUTT and antisense, AAAGUAAGGAUAGUCGCCGTT ; mouse Beclin 1-siRNA sense, GAGUUGCCGUUAUACUGUUCU and antisense, AACAGUAUAACGGCAACUCCU ; mouse LAMP-2A-siRNA sense, GAGGAGUACUUAUUCUAGUGU and antisense, ACUAGAAUAAGUACUCCUCCC ; mouse Hsc70-siRNA sense, CGAUGAAGCUGUUGCCUAUTT and antisense, AUAGGCAACAGCUUCAUCGGG; […]
Full Sequence of mt-mKeima
ATGCATCTCGAGGCCACCATGTCCGTCCTGACGCCGCTGCTGCTGCGGGGCTTGACAGGCTCGGCCCGGCGGCTCCCAGTGCCGCGCGCCAAGATCCAT TCGTTGCCGCCGGAGGGGAAGCTTGGGATGTCCGTCCTGACGCCGCTGCTGCTGCGGGGCTTGACAGGCTCGGCCCGGCGGCTCCCAGTGCCGCGCGCC AAGATCCATTCGTTGCCGCCGGAGGGGAAGCTCGGGggatccGCCATGGTGAGCGTGATCGCCAAGCAGATGACCTACAAGGTGTACATGAGCGGCACC GTGAACGGCCACTACTTCGAGGTGGAGGGCGACGGCAAGGGCAAGCCCTACGAGGGCGAGCAGACCGTGAAGCTGACCGTGACCAAGGGTGGCCCCC TGCCCTTCGCCTGGGACATCCTGAGCCCCCAGCTCCAGTACGGCAGCATCCCCTTCACCAAGTACCCCGAGGACATCCCCGACTACTTCAAGCAGAGCTT CCCCGAGGGCTACACCTGGGAGCGCAGCATGAACTTCGAGGACGGCGCCGTGTGCACCGTGAGCAACGACAGCAGCATCCAGGGCAACTGCTTCATCT ACAACGTGAAGATCAGCGGCGAGAACTTCCCCCCCAACGGCCCCGTGATGCAGAAGAAGACCCAGGGCTGGGAGCCCAGCACCGAGCGCCTGTTCGC CCGCGACGGAATGCTGATCGGCAACGACTACATGGCCCTGAAGCTGGAGGGCGGCGGCCACTACCTGTGCGAGTTCAAGAGCACCTACAAGGCCAAGA AGCCCGTGAGGATGCCCGGCCGCCACGAGATCGACCGCAAGCTGGACGTGACCAGCCACAACCGCGACTACACCAGCGTGGAGCAGTGCGAGATCGCC ATCGCCCGCCACTCCCTGCTGGGCTAA
AAV-EF1a-FRT-mCherry-WPRE and AAV-CAG-FRT-GFP-WPRE Ready to Package
AAV-EF1a-FRT-mCherry-WPRE and AAV-CAG-FRT-GFP-WPRE Ready to Package
siRNAs targeting human Caspase
Human Caspase-1 target region: AAGGGGCACAGGCATGCCAAA Human Apaf-1 target region: : AATTGGTGCACTTTTACGTGA Human Caspase-2 target region: AAACAGCTGTTGTTGAGCGA
AAV-CMV-SpCas9-HF1 (Cas9 High-fidelity Version) Ready to Package
AAV-CMV-SpCas9-HF1 (Cas9 High-fidelity Version) Ready to Package
Effective delivery of large genes to the retina by dual AAV vectors
Retinal gene therapy with adeno-associated viral (AAV) vectors is safe and effective in humans. However, AAV’s limited cargo capacity prevents its application to therapies of inherited retinal diseases due to […]