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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Cystic fibrosis: CFTR-gene editing may cure this genetic disease

August 13, 2019 – A very significant research article by the title “Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing” has just appeared in the open access Journal Nature Communications. This work provide hope to patients with cystic fibrosis (CF) that in the nearer future CF may be curable,

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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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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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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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  • Citizen science and paddle surf to study microplastic pollution in Barcelona's coastline February 27, 2020
    A team of the University of Barcelona has studied for the first time the presence of microplastics in the coasts of Barcelona, with the collaboration of the citizens gathering scientific samples. The study, published in the journal Science of Total Environment, is a pioneer citizen science study worldwide, since it analyses a hard-access area for […]
  • Celiac disease might be cured by restoring immune tolerance to gliadin February 27, 2020
    Celiac disease affects 0.3-2.4% of people in most countries world-wide, and approx. 2% in Finland. Celiac patients suffer from a variety of symptoms, typically intestinal complaints, such as diarrhea, but are often symptom-free. Immunologist Tobias Freitag co-developed and tested nanoparticles containing gliadin for the immunomodulatory treatment of celiac disease in Professor Seppo Meri's research group […]
  • Abnormal growth of bacterial cells could be linked to anti-microbial resistance February 27, 2020
    Scientists from the University of Surrey have identified mutations in a gene in an Escherichia coli (E.coli) model that could help explain a form of anti-microbial resistance (AMR) known as 'persistence'.
  • Study unravels how our immune system deals with fungal and viral infections February 27, 2020
    The body's immune response to fungal infections changes when a patient is also infected by a virus, according to new research which investigated the two types of infection together for the first time.
  • How the brain separates words from song February 27, 2020
    The perception of speech and music -- two of the most uniquely human uses of sound -- is enabled by specialized neural systems in different brain hemispheres adapted to respond differently to specific features in the acoustic structure of the song, a new study reports.
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