Neena Haider, PhD, Department of Ophthalmology, Harvard Medical School
Dr. Haider’s research is focused on promising gene-based interventions that could lead to treatments or cures for retinal disorders. The core focus of the research efforts supported by her AMDF Breakthrough Award extension, in the amount of $100,000, is developing the first broad spectrum therapy for AMD. This study is based on a recent, major breakthrough.
Rather than developing a gene-replacement or gene-editing therapy for any single gene linked to a retinal disorder, Dr. Haider has taken the approach of modifying a gene that controls an entire cascade of gene-driven events that cause a retinal disease. She showed that this could be effective for forms of retinitis pigmentosa (RP, a family of retinal disorders that is rarer than AMD, and less complex in its genetic causes), and this gene-modifier therapy approach is the first approved by the FDA.
Dr. Haider is now testing a broad spectrum treatment for AMD models of disease and initial data shows promising results that could lead to a therapy that prevents dry AMD. This work is groundbreaking as it can lead to development of prognostic tests and a treatment to prevent early stages of AMD. She is also initiating projects to develop broad spectrum therapies for aging immune-neurological diseases such as AMD and Alzheimer’s, as they share common molecular pathologies.
Dr. Haider has also discovered the first multigenic genetic model for AMD. This is unique in that it can recapitulate the molecular complexity of issues found in patients with AMD rather than targeting one gene at a time. This model will be utilized to evaluate the efficacy of current therapies developed in the Haider lab as well as to develop novel therapeutic avenues.
Olivares, A. M., Moreno-Ramos, O. A., & Haider, N. B. (2016). Role of Nuclear Receptors in Central Nervous System Development and Associated Diseases. Journal of experimental neuroscience, 9(Suppl 2), 93–121. https://doi.org/10.4137/JEN.S25480
Olivares AM, Han Y, Soto D, Flattery K, Marini J, Mollema N, Haider A, Escher P, DeAngelis MM, Haider NB. The nuclear hormone receptor gene Nr2c1 (Tr2) is a critical regulator of early retina cell patterning. Dev Biol. 2017 Sep 1;429(1):343-355. doi: 10.1016/j.ydbio.2017.05.021. Epub 2017 May 25. Erratum in: Dev Biol. 2017 Sep 1;429(1):370. PMID: 28551284.
Li, S., Datta, S., Brabbit, E., Love, Z., Woytowicz, V., Flattery, K., Capri, J., Yao, K., Wu, S., Imboden, M., Upadhyay, A., Arumugham, R., Thoreson, W. B., DeAngelis, M. M., & Haider, N. B. (2021). Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa. Gene therapy, 28(5), 223–241. https://doi.org/10.1038/s41434-020-0134-z
Iannaccone, A., Brabbit, E., Lopez-Miro, C., Love, Z., Griffiths, V., Kedrov, M., & Haider, N. B. (2021). Interspecies Correlations between Human and Mouse NR2E3-Associated Recessive Disease. Journal of clinical medicine, 10(3), 475. https://doi.org/10.3390/jcm10030475