AbstractSynthetic DNA oligonucleotides can direct the exchange of single nucleotides within coding regions of mammalian genes by hybridizing to their complementary sequence in the chromosome and creating a recombination joint structure with a single mismatched base pair. Inherent DNA repair processes recognize the mismatch and resolve it using the DNA sequence of the oligonucleotide vector as the template. This gene surgery approach can be used to repair mutations or to disrupt tri-nucleotide repeats in dysfunctional genes responsible for neurological disorders
Abstract Background Current strategies for gene therapy of inherited diseases consist in adding func...
In both replicating and non-replicating cells, the maintenance of genomic stability is of utmost imp...
Human genetic syndromes deficient in nucleotide excision repair (NER), such as xeroderma pigmentosum...
The ability to target DNA specifically at any given position within the genome allows many intriguin...
Synthetic oligonucleotides had been employed in DNA repair and promised great potentials in gene the...
Abstract Transfer of full-length genes including regulatory elements has been the preferred gene the...
Chimeric RNA/DNA oligonucleotide-based gene therapy.BackgroundChimeric RNA/DNA oligonucleotides, eme...
The major impact of the human genome sequence is the understanding of disease etiology with deduced ...
Many genetic neurological diseases result from the dysfunction of single proteins. Genetic therapies...
A number of DNA repair disorders are known to cause neurological problems. These disorders can be br...
Targeted gene modification mediated by single-stranded oligonucleotides (SSOs) holds great potential...
AbstractGene augmentation is an attractive and viable approach in treatment of inherited diseases, d...
Genome engineering using single-stranded oligonucleotides is an efficient method for generating smal...
An estimated 60% of all human genes undergo alternative splicing, a highly regulated process that pr...
Targeted gene repair mediated by single-stranded oligonucleotides (SSOs) has great potential for use...
Abstract Background Current strategies for gene therapy of inherited diseases consist in adding func...
In both replicating and non-replicating cells, the maintenance of genomic stability is of utmost imp...
Human genetic syndromes deficient in nucleotide excision repair (NER), such as xeroderma pigmentosum...
The ability to target DNA specifically at any given position within the genome allows many intriguin...
Synthetic oligonucleotides had been employed in DNA repair and promised great potentials in gene the...
Abstract Transfer of full-length genes including regulatory elements has been the preferred gene the...
Chimeric RNA/DNA oligonucleotide-based gene therapy.BackgroundChimeric RNA/DNA oligonucleotides, eme...
The major impact of the human genome sequence is the understanding of disease etiology with deduced ...
Many genetic neurological diseases result from the dysfunction of single proteins. Genetic therapies...
A number of DNA repair disorders are known to cause neurological problems. These disorders can be br...
Targeted gene modification mediated by single-stranded oligonucleotides (SSOs) holds great potential...
AbstractGene augmentation is an attractive and viable approach in treatment of inherited diseases, d...
Genome engineering using single-stranded oligonucleotides is an efficient method for generating smal...
An estimated 60% of all human genes undergo alternative splicing, a highly regulated process that pr...
Targeted gene repair mediated by single-stranded oligonucleotides (SSOs) has great potential for use...
Abstract Background Current strategies for gene therapy of inherited diseases consist in adding func...
In both replicating and non-replicating cells, the maintenance of genomic stability is of utmost imp...
Human genetic syndromes deficient in nucleotide excision repair (NER), such as xeroderma pigmentosum...