With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now found a commercialization partner for both the U.S. and Asian markets.

Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share price.

Jan. 13, 2025 — Girolline, a compound extracted from the sea sponge Pseudaxinyssa cantharella, has been investigated for possible antitumor effects and also found to have anti-malarial effects ...

The lysine methyltransferase 2B (KMT2B) gene product is important for epigenetic modifications associated with active gene transcription in normal development and in maintaining proper neural function ...

Conclusion: Although RAI1 is the primary gene responsible for most features of SMS, other genes within 17p11.2 contribute to the variable features and overall severity of the syndrome. Statistical ...

Routine genetic testing for both unilateral and bilateral cases in the clinic may increase ... Developmental delay/intellectual disability was found in eight patients, all of whom had an MRI: six ...

Germline mutations in the TP53 gene are uncommon and associated with a specific cancer syndrome known as Li-Fraumeni syndrome. People with Li-Fraumeni syndrome often develop cancer as children or ...

Although a wide variety of normal hair pigmentation exists, a number of specific findings may warrant further investigation. The examiner should be alert for areas of hypopigmentation as well as ...

Jan. 30, 2025 — A research group has developed new advanced light-controlled tools that enable precise control of proteins in real time in living cells. This groundbreaking research opens doors ...

This complex process, known as gene expression, determines which proteins we make and whether we make them in the correct amounts, crucial work given that proteins play a role in virtually every a ...