7 results about "Cftr gene" patented technology

This invention relates to the finding that novel splice switching oligonucleotides can correct splicing mutations. Moreover, the invention relates to using the novel splice switching oligonucleotides to correct a 1811+1.6kb G>T (c. 1679+1643G>T). 1811+1.6kb A>G (c. 1679+1634A>G), 1811+1.6kb T>A (c. 1679+1650T>A), or W1282X (c.3846G>A) mutation in a pre-mRNA produced from the human CFTR gene and methods of using the same for treatment of cystic fibrosis (CF) in a subject.

Provided are plasmids comprising transcriptional control elements, including transcription factor binding motifs, telomeric repeat motifs, transcription factor repressor binding motifs and promoters for tunable protein expression in target cells; wherein the plasmid for controlled transcription in a target cell type comprises a transcription factor binding motif for NFIA and the cell expresses NFIA. In one embodiment, the cell type is lung cells and the transcription factor is Nuclear Factor IA (NFIA).

The present invention relates to: a pharmaceutical composition for preventing or treating necrotic enteritis, the pharmaceutical composition containing TGF-β1 and FGF2 genes or proteins as active ingredients; and a pharmaceutical preparation and a health functional food that contain the pharmaceutical composition. According to the present invention, TGF-β1 and FGF2, which are known to increase the expression of the CFTR gene, can be used as novel agents for inducing intestinal maturation in premature infants, and various digestive complications such as necrotic enteritis and intestinal absorption disorders, which are intractable diseases, can be treated through the same.

In some aspects, provided herein are compositions, methods, and kits relating to an agent modulates expression of a CFTR protein. An agent provide herein can modify the CFTR gene or modulate process of the CFTR pre-mRNA. In some embodiments, the compositions, methods, and kits provided herein are applicable for treatment of cystic fibrosis.

The present invention relates to the field of biotechnology, genetic engineering and medicine, in particular, to a highly efficient system for editing the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene to the wild type, comprising a polynucleotide which comprises a nucleotide sequence, encoding the SpCas9 nickase recognizing PAM NGG or NG, and a prime editing guide RNA (pegRNA) comprising in its structure a sequence complementary to the target locus for editing the F508del mutation in human CFTR gene, a reverse transcription template (RTT) and a primer binding site (PBS), wherein the prime editing guide RNA (pegRNA) has sequence SEQ ID NO: 1-6 or a sequence comprising one or more replacements in the RTT compared to SEQ ID NO: 1-6, selected from SEQ ID NO: 7-168. The system for editing the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene to the wild type provides more efficient editing of the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene to the wild type and is a highly efficient cystic fibrosis treatment. The present invention also relates to the method of editing the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene using the system for editing the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene according to the present invention and the use of the system for editing the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene for treating cystic fibrosis.

Are disclosed pharmaceutical compositions comprising as active ingredients Esculentin-1a(1-21)NH2 and / or Esculentin diastereomer Esc(1-21)-1c for use for restoring dysregulation of water and / or ions content and / or composition of the periciliary liquid due to mutations of the CFTR gene encoding for Cystic fibrosis transmembrane conductance regulator (CFTR) and for use for the treatment of cystic fibrosis.