Blog Archives

Gene therapy: Zolgensma to treat spinal muscular atrophy

May 29, 2019 – Spinal muscular atrophy (SMA) is a group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting. The severity of symptoms and age of onset varies by the type. Some types are apparent at or before birth while others are not apparent until adulthood. 

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CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy: Prime time already?

December 12, 2018 – The application of CRISPR/Cas9 based molecular technology in the field of gene editing (or genome editing) has recently had its exploded limelight exposure for a couple of reasons. The question arises if this exposure is earned or somewhat premature.

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Voretigene Neparvovec-Rzyl (Luxturna): Gene therapy to treat inherited vision loss

December 20, 2017 –  The American Food & Drug Administration (FDA) has just approved  a novel gene therapy to treat patients with a rare form of inherited vision loss. Voretigene Neparvovec-Rzyl (Luxturna) is the first directly administered gene therapy approved to target a disease caused by mutations in a specific human gene (i.e.,

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Spinal Muscular Atrophy: Single-Dose Gene-Replacement Therapy

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Living theragenomics: Dangers of codeine and tramadol medicines in children

April 23, 2017 – This is a Safety Announcement from April 20, 2017, by the American Food & Drug Administration (FDA) to the fact that FDA further restricts use of prescription codeine pain and cough medicines and tramadol pain medicines in children and recommends against their use in breastfeeding women.

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Children: Is there personalized medicine for children too?

April 04, 2017 – Personalized medicine, also referred to as precision medicine, incorporates the individual patient’s characteristics into treatment, rather than relying on population means. Over the past several years, it has become a significant focus for research. Developments in pharmacogenomics, the study of genomic variations that influence response to drugs,

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  • MicroRNA exhibit unexpected function in driving cancer February 20, 2020
    Researchers long thought that only one strand of a double-stranded microRNA can silence genes. Though recent evidence has challenged that dogma, it's unclear what the other strand does, and how the two may be involved in cancer. New research from Thomas Jefferson University has revealed that both strands of some microRNA coordinate to act on […]
  • New therapy stops seizures in mouse model of rare childhood epilepsy February 20, 2020
    Seizure disorders in babies are frightening and heartbreaking. A new basic science breakthrough offers hope for a potential treatment for rare developmental and epileptic encephalopathies resulting from a single genetic mutation. The gene in question, called SCN8A, controls a sodium channel that allows neurons to transmit an electric signal. When this gene is mutated, these […]
  • Scientists find many gene 'drivers' of cancer, but warn: Don't ignore 'passengers' February 20, 2020
    A massive analysis of the entire genomes of 2,658 people with 38 different types of cancer has identified mutations in 179 genes and gene regulators as "drivers"—variations in DNA sequences that lead to the development of cancer.
  • A deep dive into cellular aging February 20, 2020
    Scientists at Sanford Burnham Prebys Medical Discovery Institute and Harvard University have discovered that mitochondria trigger senescence, the sleep-like state of aged cells, through communication with the cell's nucleus—and identified an FDA-approved drug that helped suppress the damaging effects of the condition in cells and mice. The discovery, published in Genes & Development, could lead […]
  • New model of C. elegans helps progress study of rare genetic disease February 20, 2020
    The IDIBELL Neurometabolic Diseases group, with international collaboration, has identified a model of chromosome X-linked adrenoleukodystrophy (X-ALD) in C. elegans. X-ALD is a rare disorder of the nervous system with no available treatment.
  • Half of transgender youth avoid disclosing gender identity to a health care provider February 20, 2020
    Researchers surveyed 153 transgender youths receiving gender-affirming care at a specialty clinic, and even in this relatively 'out' population, nearly half reported intentionally hiding their gender identity from a health care provider outside the clinic.
  • Fifty years of data show new changes in bird migration February 20, 2020
    A growing body of research shows that birds' spring migration has been getting earlier and earlier in recent decades. New research from The Auk: Ornithological Advances on Black-throated Blue Warblers, a common songbird that migrates from Canada and the eastern US to Central America and back every year, uses fifty years of bird-banding data to […]
  • New study supports the safety of varenicline February 20, 2020
    A real-world study of over 600,000 adult participants without a history of depression has found that the stop-smoking medication varenicline (marketed as Chantix and Champix) does not appear to be associated with an increased risk of cardiovascular or neuropsychiatric hospitalization compared with nicotine replacement therapy (NRT). These findings confirm those of earlier clinical trials, providing […]
  • New study indicates amino acid may be useful in treating ALS February 20, 2020
    A naturally occurring amino acid is gaining attention as a possible treatment for ALS following a new study published in the Journal of Neuropathology & Experimental Neurology. It showed the amino acid, L-serine, successfully reduced ALS-like changes in an animal model of ALS. After exposure to the cyanobacterial neurotoxin, BMAA, vervets developed pathology similar to […]
  • Patients frequently refuse insulin therapy, delaying blood sugar control February 20, 2020
    Investigators from Brigham and Women's Hospital find that more than 40% of patients refuse a physician's recommendation of insulin therapy. The study also finds that patients who decline insulin therapy had worse blood sugar control and it took them significantly longer to lower their blood sugar levels than patients who began insulin therapy.
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