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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 311
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Nanorobot hand made of DNA grabs viruses for diagnostics and blocks cell entry

Science Daily: Pharmacology News

A tiny, four-fingered 'hand' folded from a single piece of DNA can pick up the virus that causes COVID-19 for highly sensitive rapid detection and can even block viral particles from entering cells to infect them, researchers report.

DNA 299
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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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Researchers map communities of single cells in metastatic breast cancers

Broad Institute

Researchers map communities of single cells in metastatic breast cancers By Corie Lok October 30, 2024 Breadcrumb Home Researchers map communities of single cells in metastatic breast cancers The team compared different single-cell and spatial profiling methods to explore the diversity of cells in metastatic breast cancer biopsies.

Research 127
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Bridging Innovation & Patient Care: The Growing Role of AI

Speaker: Simran Kaur, Co-founder & CEO at Tattva.Health

But its impact goes far beyond research. From Research to Real-World Care 🌎 Understand how AI-driven advancements are shaping personalized treatments and patient outcomes. AI is transforming clinical trials—accelerating drug discovery, optimizing patient recruitment, and improving data analysis.

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With AI, researchers predict the location of virtually any protein within a human cell

Broad Institute

With AI, researchers predict the location of virtually any protein within a human cell By Corie Lok May 19, 2025 Breadcrumb Home With AI, researchers predict the location of virtually any protein within a human cell Trained with a joint understanding of protein and cell behavior, the model could help with diagnosing disease and developing new drugs.

Research 105
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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

Alltrna’s unique approach focuses on addressing genetic diseases at a fundamental level, shifting the paradigm from a one-disease, one-therapy model to a mutation-specific strategy that could transform the treatment of hundreds of rare diseases. This will mark a critical step in validating the potential of engineered tRNA therapeutics.

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