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Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment

Science Daily: Pharmacology News

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.

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Novel stem cell therapy repairs 'irreversible' corneal damage in clinical trial

Science Daily: Pharmacology News

Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.

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FDA meeting gives window into gene therapy field’s angst

BioPharma Drive: Drug Pricing

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a regulatory forum Thursday.

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Deafness reversed: Single injection brings hearing back within weeks

Science Daily: Pharmacology News

A cutting-edge gene therapy has significantly restored hearing in children and adults with congenital deafness, showing dramatic results just one month after a single injection. The therapy worked best in young children but still benefited adults, with one 7-year-old girl regaining almost full hearing.

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AGPAT4 targeted covalent inhibitor potentiates targeted therapy to overcome cancer cell plasticity in hepatocellular carcinoma mouse models

Covalent Modifiers

Tumor lineage plasticity has been identified as a key factor in therapy resistance and tumor recurrence. In conclusion, the plasticity of tumor lineages induced by AGPAT4 represents a potential target for HCC treatment and could expand the effectiveness of sorafenib treatment, offering new possibilities for HCC therapy.

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Zebrafish protein unlocks dormant genes for heart repair

Science Daily: Pharmacology News

This study marks an important step toward regenerative therapies to prevent heart failure. In mice, this protein was able to restore the heart by activating dormant repair genes without causing side effects, such as heart enlargement.

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CRISPR uncovers gene that supercharges vitamin D—and stops tumors in their tracks

Science Daily: Pharmacology News

This opens the door to highly targeted cancer therapies—by either cutting off vitamin D supply to tumors or enhancing the gene’s activity to boost health. Researchers found that disabling this gene in colorectal cancer cells not only crippled their survival but also disrupted thousands of other genes tied to cancer and metabolism.