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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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  • Descendants of early Europeans and Africans in US carry Native American genetic legacy September 19, 2019
    Many people in the U.S. do not belong to Native American communities but still carry bits of Native American DNA, inherited from European and African ancestors who had children with indigenous individuals during colonization and settlement. In a new study published 19th September in PLOS Genetics, Andrew Conley of the Georgia Institute of Technology and […]
  • IGF1 gene is essential to adult tendon growth, animal study shows September 19, 2019
    Tendon injuries are among the most common injuries seen in athletes at all levels, from weekend warriors to professional basketball players. For those who rupture their tendons, returning to the same level of physical activity they enjoyed before the injury is rare.
  • Researchers create new protocol to improve gene therapy tool production September 19, 2019
    A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers.
  • Genetic variants with possible positive implications for lifestyle September 19, 2019
    A German and British research team lead by Technical University of Munich (TUM) has examined the interplay between genetics, cardiovascular disease and educational attainment in a major population study. Genetic variants which had been linked to educational attainment in other studies were observed in the subjects. The researchers found that these variants also had implications […]
  • Scientists develop new methodology to genetically modify lab mice and human cells September 19, 2019
    A team led by Cedars-Sinai has designed a rapid method to genetically alter laboratory mice and then used this method to produce personalized animal models of pediatric glioma, an aggressive type of malignant brain cancer in children.
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