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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  • Artificial RNA editing with ADAR for gene therapy July 9, 2020
    Many of the diseases caused by point mutations have no established therapeutic approaches. Prof. Tsukahara and colleagues (Japan Advanced Institute of Science and Technology) are studying a therapeutic method using artificial RNA editing. Artificial site-directed RNA editing is an important technique for modifying genes and ultimately regulating protein function. We are trying to modify the […]
  • Amygdala changes in male patients with schizophrenia and bipolar disorder July 9, 2020
    Researchers at Kumamoto University, Japan have revealed that DNA methylation occurs in the gene that codes serotonin transporter (SERT), a protein that regulates neurotransmitter transmission, in schizophrenia and bipolar patients. Particularly prominent in males and patients with certain genetic polymorphisms, this methylation is inversely correlated with volume of the amygdala in the brain. This work […]
  • Surprisingly many peculiar long introns found in brain genes July 9, 2020
    In a recent study of genes involved in brain functioning, their previously unknown features have been uncovered by bioinformaticians from the Moscow Institute of Physics and Technology and the Institute of Mathematical Problems of Biology, RAS. The findings are reported in PLOS One.
  • Major cause of rare genetic mitochondrial disease identified July 9, 2020
    A cutting-edge study from the Murdoch Children's Research Institute (MCRI) has given hope to families of children born with a fatal heart muscle disease caused by faulty cell machinery.
  • Spatial mapping method pinpoints potential new therapeutic targets in lupus July 8, 2020
    A team of researchers from Children's Hospital of Philadelphia (CHOP) used a new method of pinpointing potential disease-causing changes in the genome to identify two new potential therapeutic targets for lupus, while also paving the way for more accurately identifying disease-causing variations in other autoimmune disorders. The findings were published online in Nature Communications.
  • Lung screening bus brings high-tech health care directly to patients July 14, 2020
    A mobile platform for lung cancer screening with low-dose computed tomography (CT) can be developed with limited financial risk and take powerful screening tests directly to patients, including underserved rural areas where rates of new lung cancer cases tend to be higher.
  • Mismatched caregiver-infant interactions during feeding could boost babies' risk of later obesity July 14, 2020
    A new integrative review examined evidence related to infants' self-regulation of behavior and emotion, and how that relates to interactions when they are fed by their caregivers, including how those interactions may derail infants' ability to regulate their intake of food. The review found that infants who are fed in the absence of hunger or […]
  • When a pandemic strikes, we still expect an ambulance July 14, 2020
    Edith Cowan University (ECU) PhD candidate and paramedic Cameron Anderson investigated community attitudes regarding the professional obligation of paramedics to respond during pandemics.The research showed that, pandemic or not, Australians expected an ambulance to arrive if children were involved, if there was adequate protective equipment and if it involved our immediate families.
  • Age of sexual debut among young gay-identified sexual minority men July 14, 2020
    Young gay sexual minority men - especially Black and Latino youth - have their first sexual experiences at younger ages, emphasizing a need for comprehensive and inclusive sex education, according to Rutgers researchers.
  • COVID-19 pandemic could be learning opportunity for middle-grade students July 14, 2020
    Educators could use the COVID-19 outbreak to help middle-schoolers better understand the world, according to new research from faculty at Binghamton University, State University of New York.
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