the glocalized network in theragenomic and personalized medicine and the genetics of individual patients safety of drugs
September 16, 2022 – Beta thalassemias (β thalassemias) are a group of inherited blood disorders. They are forms of thalassemia caused by reduced or absent synthesis of the beta chains of hemoglobin that result in variable outcomes ranging from severe anemia …
Gene therapy: Hope for patients with beta-thalassemia? Read more »
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 …
Onasemnogene abeparvovec-xioi (Zolgensma): Approval strategy with manipulated data? Read more »
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: CFTR-gene editing may cure this genetic disease Read more »
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 …
CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy: Prime time already? Read more »
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 …
Voretigene Neparvovec-Rzyl (Luxturna): Gene therapy to treat inherited vision loss Read more »
November 12, 2017 – Spinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor neuron disease with an onset during infancy that results in failure to achieve motor milestones and in death or the need for mechanical ventilation by …
Spinal Muscular Atrophy: Single-Dose Gene-Replacement Therapy Read more »
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 …
Axicabtagene Ciloleucel (Yescarta) gene therapy for diffuse large B-cell lymphoma Read more »
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 …