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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  • Researchers identify new gene mutation in familial thyroid cancers November 20, 2019
    Researchers from Penn State College of Medicine identified a new gene mutation that may cause a type of familial thyroid cancer. Dr. Darrin Bann, an otolaryngology resident at the College of Medicine and lead author of the study, said that this mutation is the first and only mutation associated with familial thyroid cancer to be […]
  • Researchers develop new database of druggable fusion targets November 20, 2019
    When sections from two separate genes merge due to various factors, such as translocation or splicing, the hybrid that is formed is called a gene fusion. In recent years, it has been discovered that these fusion events play a vital role in the development of cancers and other complex diseases. However, there are very few […]
  • Early results of CRISPR gene-editing treatment shows promise in first human trials November 20, 2019
    Officials from Vertex Pharmaceuticals and CRISPR Therapeutics have announced that preliminary results from testing CRISPR gene-editing treatment in human patients with blood disorders show promise thus far. The joint project between the two firms is taking place at one location in Europe and another in the United States. The results have been posted on the […]
  • Whole-genome sequencing analysis to find more exact biomarkers November 20, 2019
    A new study from Uppsala University shows that whole-genome sequencing increases the precision of genetic studies, which in turn can improve our understanding of how to use biomarkers to discover disease. The results are published in the scientific journal Scientific Reports.
  • Complex organ models grown in the lab November 19, 2019
    Scientists at the University of Würzburg have successfully produced human tissues from stem cells. They have a complexity similar to that of normal tissue and are far superior to previous structures.
  • New study confirms American children and teens are consuming significantly less sugary drinks November 21, 2019
    According to a new study in the American Journal of Preventive Medicine, published by Elsevier, the share of children and adolescents consuming sugar-sweetened beverages (SSBs) and the calories they consume from SSBs declined significantly between 2003 and 2014.
  • New Alzheimer risk gene discovered November 21, 2019
    A new paper in the Journal of Neuropathology & Experimental Neurology finds a gene that may help explain a large part of the genetic risk for developing Alzheimer disease.
  • New type of e-cigarette vaping injury described in CMAJ November 21, 2019
    A research case report describing lung injury related to e-cigarette use in a 17-year-old Canadian may be the first documented case of a new form of damage from vaping products. The article, published in CMAJ (Canadian Medical Association Journal) provides new evidence on forms of lung injury that can result from vaping.
  • CUHK Faculty of Engineering develops novel imaging approach November 21, 2019
    By combining a compressive sensing algorithm with a digital holographic microscope, Prof. Shih-Chi CHEN of the Department of Mechanical and Automation Engineering, Faculty of Engineering, The Chinese University of Hong Kong (CUHK) and his research team have developed a high-speed imaging method. The new approach is able to produce two-photon microscopy images of a 3D […]
  • Cybershoppers make better buying decisions on PCs than phones -- Ben-Gurion U. researchers November 21, 2019
    This is the first study that differentiates between screen size and information reduction, which are often mixed up. The findings will be presented next month at the International Conference on Information Systems, the top academic conference in the field.
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