Rebecca C. Ahrens‐Nicklas

Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

Kiran Musunuru, Sarah Grandinette, Xiao Wang et al. · New England Journal of Medicine · 2025 · 179 citations · Full text

Genetic Diseases, Genome Surgery, In Vivo Gene Therapy +15

Stimulation of entorhinal cortex–dentate gyrus circuitry is antidepressive

Sanghee Yun, Ryan Reynolds, Iraklis Petrof et al. · Nature Medicine · 2018 · 105 citations · Full text

Psychiatry, Neurophysiology, Neuroanatomy +9

Complex care of individuals with multiple sulfatase deficiency: Clinical cases and consensus statement

Rebecca C. Ahrens‐Nicklas, Lars Schlotawa, Andrea Ballabio et al. · Molecular Genetics and Metabolism · 2018 · 50 citations

Complex Care, Inherited Metabolic Disease, Multiple Sulfatase Deficiency +3

Precision therapy for a new disorder of AMPA receptor recycling due to mutations in <i>ATAD1</i>

Rebecca C. Ahrens‐Nicklas, George K. E. Umanah, Neal Sondheimer et al. · Neurology Genetics · 2017 · 49 citations · Full text

Pathologic Variants of the Mitochondrial Phosphate Carrier SLC25A3: Two New Patients and Expansion of the Cardiomyopathy/Skeletal Myopathy Phenotype With and Without Lactic Acidosis

Elizabeth Bhoj, M. Li, Rebecca C. Ahrens‐Nicklas et al. · JIMD Reports · 2014 · 48 citations · Full text