• Mashup Score: 8

    In this review, Davidson and colleague provide an in-depth review of the preclinical and clinical gene therapy advances made in treatment of both inherited and sporadic amyotrophic lateral sclerosis (ALS). They discuss antisense oligonucleotides, RNA interference, CRISPR, AAV-mediated trophic support, and antibody-based methods, highlighting ongoing challenges and anticipated future directions.

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    • Most-read Molecular Therapy paper: Gene Therapy for ALS: A Review @ASGCTherapy @MolTherapy @lab_davidson @PennMedicine https://t.co/xb0Xku0vjL

  • Mashup Score: 0

    The introduction of chimeric antigen receptor T cell (CAR-T) therapy has been transformative in hematological malignancies. In the US, there are now 6 FDA-approved products across 5 malignancies; acute lymphoblastic leukemia, diffuse large B cell lymphoma (DLBCL), follicular lymphoma, mantle cell lymphoma, and multiple myeloma. These are highly potent treatments that are effective in patients…

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    • Great collaboration with @MichaelDJain and @lisa_masucci - our opinion peace in Molecular Therapy re: the high cost of cellular therapies. https://t.co/EmoNt7fb9p

  • Mashup Score: 5

    Researchers have been trying to develop therapies for hereditary diseases for more than 30 years. Several problems have been successfully resolved and thus Tremblay and colleagues are describing the recent progress of clinical applications of gene therapy. There are now several in vivo treatments under trials, and some approved for commercialization.

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    • Most-read Molecular Therapy paper: Current Clinical Applications of in vivo Gene Therapy with AAVs @ASGCTherapy @MolTherapy @CIHR_IRSC https://t.co/DbWgPX1z8W