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Researchers grow 400+ brain cell types—a leap for Alzheimer’s and Parkinson’s research

Science Daily: Pharmacology News

By systematically experimenting with combinations of morphogens and gene regulators, the researchers replicated the vast diversity of neurons found in the human brain.

Research 260
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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.

Treatment 278
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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. Researchers used a virus to deliver a healthy copy of the OTOF gene into the inner ear, improving auditory function across all ten participants in the study.

Virus 279
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Novel stem cell therapy repairs 'irreversible' corneal damage in clinical trial

Science Daily: Pharmacology News

The groundbreaking procedure was developed to help people with injuries like chemical burns that lead to irreversible limbal stem cell deficiency, and the researchers hope the new findings warrant additional trials.

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Zebrafish protein unlocks dormant genes for heart repair

Science Daily: Pharmacology News

Researchers have successfully repaired damaged mouse hearts using a protein from zebrafish. This study marks an important step toward regenerative therapies to prevent heart failure. This study marks an important step toward regenerative therapies to prevent heart failure.

Therapies 289
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Researchers use machine learning to engineer 'bespoke enzymes' for gene editing

Science Daily: Pharmacology News

A new paper showcases the power of scalable protein engineering combined with machine learning to boost progress in the field of gene and cell therapy. The work could help reduce off-target effects and improve editing safety, enhance editing efficiency, and enable researchers to predict customized enzymes for new therapeutic targets.

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

Science Daily: Pharmacology News

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. 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.

Therapies 216