Alexandra Zezulin

Journal article Genes & development · July 2023 The transcription factor RUNX1 is mutated in familial platelet disorder with associated myeloid malignancy (FPDMM) and in sporadic myelodysplastic syndrome and leukemia. RUNX1 was shown to regulate inflammation in multiple cell types. Here we show that RUN ... Full text Cite

Inclusion of a short hairpin RNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin.

Journal article Haematologica · October 2021 Full text Cite

AKT3 Gene Transfer Promotes Anabolic Reprogramming and Photoreceptor Neuroprotection in a Pre-clinical Model of Retinitis Pigmentosa.

Journal article Molecular therapy : the journal of the American Society of Gene Therapy · July 2019 Mutations within over 250 known genes are associated with inherited retinal degeneration. Clinical success following gene-replacement therapy for congenital blindness due to RPE65 mutations establishes a platform for the development of downstream treatment ... Full text Cite

SIRT1 and NRF2 Gene Transfer Mediate Distinct Neuroprotective Effects Upon Retinal Ganglion Cell Survival and Function in Experimental Optic Neuritis.

Journal article Investigative ophthalmology & visual science · March 2018 PurposeOptic neuritis is a condition defined by autoimmune-mediated demyelination of the optic nerve and death of retinal ganglion cells. SIRT1 and NRF2 stimulate anti-inflammatory mechanisms and have previously demonstrated therapeutic value in p ... Full text Cite

Turning up the Heat with Therapeutic Epigenome Editing.

Other Cell stem cell · January 2018 CRISPR-Cas9 has been touted for therapeutic genome editing, but limitations include inefficient correction of disease-causing mutations and off-target mutagenesis. In the latest issue of Cell, Liao et al. (2017) show that a modified version of CRISPR-Cas9 ... Full text Cite