Gene Therapy
Modifying genes to treat diseases by introducing new genetic materials, altering existing genes, or silencing harmful genes.
* ASO (Antisense oligonucleotide), siRNA, CRISPR/Cas9, and Aptamer
Drug discovery for gene therapy
* ASO (Antisense oligonucleotide), siRNA, CRISPR/Cas9, and Aptamer
*The cells in the body may have immune responses that recognize the therapeutic gene as foreign and may attack them, which can lead to ineffective delivery and potential harm to the patient.
*Modifying the structure of RNA or DNA molecules, such as adding or removing certain chemical groups, in order to improve their properties.
