Bespoke CRISPR Lifeline: First-in-World Gene Editing Treats Infant’s CPS1 Deficiency

When baby KJ was diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency—a rare metabolic disorder that can trigger life-threatening ammonia buildup, his family faced a narrow path: strict dietary…

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 Mitochondrial Dysfunction is Identified by Researchers Seeking a Common Denominator for Gene Mutations Believed to Cause ALS

Researchers at the UK Dementia Institute in London (UK DRI) have identified a common denominator for various gene mutations that cause amyotrophic lateral sclerosis (ALS). A new CRISPR study shed…

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U.K. Health Authorities Grant Approval for a New Medicine using CRISPR Gene Editing to Treat Beta thalassemia and Sickle Cell
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U.K. Health Authorities Grant Approval for a New Medicine using CRISPR Gene Editing to Treat Beta thalassemia and Sickle Cell

CRISPR was first described ten years ago as a viral mechanism for defense, taken from bacteria, which could be transformed into genetic scissors and accurately alter DNA. About Casgevy CRISPR…

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Scientists May Have Found a Possible Cure for Acute Leukemia in Base Edited T-Cells
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Scientists May Have Found a Possible Cure for Acute Leukemia in Base Edited T-Cells

Leukemia is cancer that begins in the bone marrow and involves abnormal white blood cells (WCB) from lymphatic tissues and bone marrow. Scientists have new theories as to why certain…

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The FDA Grants Approval to Begin Dosing First-in-Human CRISPR Technology to Treat Duchenne Muscular Dystrophy
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The FDA Grants Approval to Begin Dosing First-in-Human CRISPR Technology to Treat Duchenne Muscular Dystrophy

   Business Wire published an August 10th news release announcing that Cure Rare Disease, a non-profit based in Boston, Massachusetts received FDA approval to administer CRD-TMH-001 (CRD), its first-ever therapeutic.…

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CRISPR Continues to Cut Impressive Numbers in Beta-Thalassemia, Sickle Cell Disease, and More

A year ago, Paddy Doherty’s doctor told him that he had a rare hereditary disease called transthyretin (ATTR) amyloidosis, the same disease that had killed his father. As reported in…

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Patient Worthy’s One-Year-Checkup on Victoria Gray, the First Sickle Cell Disease Patient to Receive CRISPR in the US
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Patient Worthy’s One-Year-Checkup on Victoria Gray, the First Sickle Cell Disease Patient to Receive CRISPR in the US

   CRISPR is a gene-editing technique that has revolutionized the medical world with its approach to making precise changes in DNA. CRISPR found its match in 35-year-old Victoria Gray of…

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CRISPR: It Has Only Just Begun
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CRISPR: It Has Only Just Begun

  Over ten years ago two Nobel-Prize-winning biologists, Emmanuelle Charpentier and Jennifer Doudna, discovered a technology called CRISPR (clustered regularly interspaced short palindromic repeats). Bacteria contain genetic sequences repeated throughout…

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