Blog Archives

Onasemnogene abeparvovec-xioi (Zolgensma): Approval strategy with manipulated data?

August 13, 2019 – Onasemnogene abeparvovec-xioi (Zolgensma) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

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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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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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Marsili Syndrome: A mutation in the ZFHX2 gene leaves Italian family painless

December 21, 2017 – No pain on gain. This headline might not at all apply for this Italian family. In a paper just published in the Journal Brain,

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Spinal Muscular Atrophy: Single-Dose Gene-Replacement Therapy

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The first gene therapy FDA approved in the US

September 3, 2017 – For a very first time, the American Food & Drug Administration (FDA) has approved a gene therapy for the United States (US) in that it approved CAR T-cell therapy to treat certain children and young adults with B-cell acute lymphoblastic leukemia (B-cell ALL).

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Axicabtagene Ciloleucel (Yescarta) gene therapy for diffuse large B-cell lymphoma

October 19, 2017 – The American  Food & Drug Administration (FDA) just approved Axicabtagene Ciloleucel (Yescarta), a cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment.

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New hope for children with the ultra-rare immune disorder ADA-SCID: Gene therapy Strimvelis recommended for approval by EMA’s CHMP

April 02, 2016 – The European Medicines Agency (EMA) has recommended granting a marketing authorisation in the European Union (EU) for a new gene therapy for the treatment of patients with adenosine-deaminase-deficient severe combined immunodeficiency (ADA-SCID), who have no matching donor for a stem cell transplant.

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  • Study reveals restoration of retinal and visual function following gene therapy October 19, 2020
    A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.
  • Scientists map the human proteome October 19, 2020
    Twenty years after the release of the human genome, the genetic "blueprint" of human life, an international research team, including the University of British Columbia's Chris Overall, has now mapped the first draft sequence of the human proteome.
  • The line of succession in neuron function October 19, 2020
    A specific region of messenger RNAs, the 3' untranslated region (3'UTR), plays an important role for cells to function properly. During embryonic development, 3'UTRs in hundreds of RNAs lengthen exclusively in neurons, which is crucial for the cells of the brain to function properly. The lab of Valérie Hilgers at the Max Planck Institute of […]
  • Pinpointing the 'silent' mutations that gave the coronavirus an evolutionary edge October 16, 2020
    We know that the coronavirus behind the COVID-19 crisis lived harmlessly in bats and other wildlife before it jumped the species barrier and spilled over to humans.
  • Transposons could be rewiring our brains October 16, 2020
    A new study by neuroscientists at the University of Oxford shows that mobile genetic elements that were active in the genomes of our ancestors could be closely linked to important functions in our brain and might help diversify our behavior, cognition and emotions.
  • Asymmetric optical camouflage: Tuneable reflective color accompanied by optical Janus effect October 20, 2020
    Deliverying viewing-direction sensitive information display across single sheet of transreflective window is introduced. Based on the experimental verification of theoretical modelling, scientists in Republic of Korea invented colour tuneable optical device that displays different colours and messages depending on viewing direction which is completely new and exotic optical phenomenon. A step further, they realized asymmetric […]
  • Researchers at the forefront of developing machine learning methods for chemical discovery October 20, 2020
    Prof. Alexandre Tkatchenko and his research team at the University of Luxembourg have been awarded grants totalling 500,000 euros to conduct research in the emerging field of machine learning methods for chemical discoveries.
  • COVID-19: Distancing and masks are not enough October 20, 2020
    Decades-old data is being used to describe the propagation of tiny droplets. Now a fluid dynamics team has developed new models: Masks and distancing are good, but not enough. Even with a mask, infectious droplets can be transmitted over several meters and remain in the air longer than previously thought.
  • Oncotarget: Inhibition of HAS2 and hyaluronic acid production by 1,25-Dihydroxyvitamin D3 in breast October 20, 2020
    Oncotarget Volume 11, Issue 30 reported that genomic profiling of murine mammary tumor cells with differential VDR expression identified 35 transcripts that were altered by the 1,25D3-VDR complex including Hyaluronan Synthase-2.
  • US adults' likelihood of accepting COVID-19 vaccination October 20, 2020
    In this survey study of U.S. adults, vaccine-related attributes and political characteristics were associated with self-reported preferences for choosing a hypothetical COVID-19 vaccine and self- reported willingness to receive vaccination. These results may help inform public health campaigns to address vaccine hesitancy when a COVID-19 vaccine becomes available.
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