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BioWorld - Friday, May 8, 2026

Genetic/congenital

Home » Topics » Disease categories and therapies » Genetic/congenital
  • Cell gene therapies conceptual illustration
    May 7, 2026

    Engineered programmable inhibitory binders to target CRAC channelopathies

    CRAC channels are essential for immune and developmental processes, and dysregulation of store-operated Ca2+ entry (SOCE) has been implicated in several human diseases. Researchers from Texas A&M University and collaborators recently described the engineering of genetically encoded CRAC channel inhibitory binders (CRABs) derived from the ORAI C-terminal region, a defined STIM1-binding interface.
  • Brain dna pills
    May 6, 2026

    Elaaj Bio advances gene therapy for CDKL5 deficiency disorder

    Elaaj Bio, a wholly owned subsidiary of the nonprofit Loulou Foundation, has entered into a partnership with Viralgen Vector Core SL to advance a gene therapy program for CDKL5 deficiency disorder.
  • Brain genetics
    May 4, 2026

    AAV9-delivered AntagoNATs have preclinical efficacy as one-time treatment for Dravet syndrome

    Dravet syndrome is a rare, severe, lifelong developmental and epileptic encephalopathy that begins in infancy and is marked by prolonged, often fever-triggered seizures that are difficult to control. It is usually caused by mutations in the SCN1A gene and is associated with developmental delay, cognitive and behavioral impairment, and reduced life expectancy.
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Authors

  • Anette Breindl
  • Mar de Miguel
  • Helen Albert
  • Subhasree Nag
  • Xavier Bofill Bruna
  • Nuala Moran
More Authors
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