Patent ID: 11952585
Assignee: HOMOLOGY MEDICINES, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 1:
2. The method of claim 1, wherein:
the transcriptional regulatory element is capable of mediating transcription in a cell of the liver, the kidney, the brain, the pituitary gland, the adrenal gland, the pancreas, the urinary bladder, the gallbladder, the colon, the small intestine, or the breast;
the transcriptional regulatory element comprises a human hepatic control region 1 (HCR1) comprising the nucleotide sequence set forth in SEQ ID NO:2;
the transcriptional regulatory element comprises a human al-antitrypsin (hAAT) promoter comprising the nucleotide sequence set forth in SEQ ID NO:3;
the transcriptional regulatory element comprises an SV40 intron comprising the nucleotide sequence set forth in SEQ ID NO:4; or
the transcriptional regulatory element comprises the nucleotide sequence set forth in SEQ ID NO:5.