Kearns-Sayre syndrome appears due to the lack of a discrete part of mitochondrial DNA(NB: the lack of a certain nucleotide sequence is called deletion).
The distribution of these variations differed between hESCs and hiPSCs, characterized by large numbers of duplications found in a few hESC samples and moderate numbers of deletions distributed across many hiPSC samples.
Therefore, the probes of the subject invention include mutations(both single and multiple), deletions, insertions of the described sequences, and combinations thereof, wherein said mutations, insertions and deletions permit formation of stable hybrids with the target polynucleotide of interest.
Furthermore, the aforementioned substitutions, deletions, insertions, additions, inversions, or the like, of amino acid residues include naturally occurring mutations(mutant or variant), such as mutations based on individual differences and/or species differences of the microorganism having the target gene.
When comparing two amino acid sequences, the term"amino acid difference" refers to an insertion, deletion or substitution of a single amino acid residue on a position of the first sequence, compared to the second sequence;
Therefore, the probes of the subject invention include mutations(both single and multiple), deletions, insertions of the described sequences, and combinations thereof, wherein said mutations, insertions and deletions permit formation of stable hybrids with the target polynucleotide of interest.
Therefore, the probe sequences of the subject invention include mutations(both single and multiple), deletions, insertions of the described sequences, and combinations thereof, wherein said mutations, insertions and deletions permit formation of stable hybrids with the target polynucleotide of interest.
However, when used to correct a single nucleotide, the standard CRISPR/Cas9 method is usually inefficient, and frequently introduces random insertions or deletions(collectively known as indels) at the target location, mainly as a result of the cellular response to a double-stranded DNA break.
In addition to t(4;11) and t(9;22), compared with patients with a normal karyotype, patients with deletion of chromosome 7 or trisomy 8 have been reported to have a lower probability of survival at 5 years.
Studying mouse and human laboratory cell lines, Allan Bradley and colleagues find that, in addition to the small DNA errors that are known to accompany the repair of double-strand breaks, CRISPR-Cas9 can cause large deletions and, in some cases, complex rearrangements around the target site.
The biology of the adenoviruses is characterized in detail, the adenovirus is not associated with severe human pathology, the virus is extremely efficient in introducing its DNA into the host cell, the virus can infect a wide variety of cells and has a broad host range, the virus can be produced in large quantities with relative ease, and the virus can be rendered replication detective by deletions in the early region 1(“E I”) of the viral genome.
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