Fengfeng Bei, PhD
Assistant Professor
Department of Neurosurgery
Brigham and Women’s Hospital
Harvard Medical School
RESEARCH FOCUS
We are interested in gene therapy and are studying adeno-associated viruses (AAVs) as vectors for treating diseases of the brain, spinal cord, eye, and kidney. A major focus of our research is engineering new safe and effective AAV vectors to overcome gene delivery barriers. Using these optimized AAVs as tools, we aim to develop gene therapies for both rare genetic diseases and more common conditions. In addition, we are exploring complementary gene therapy technologies, including focused ultrasound-mediated drug delivery, gene switches, and RNA interference.
Through collaboration, we are currently working on:
- Spinal muscular atrophy with progressive myoclonic epilepsy (SMA-PME)
- Farber disease
- CDKL5 deficiency disorder
- Fragile X syndrome
- Mucolipidosis type IV
- Brain metastatic cancers
- Glioma
- Pulmonary fibrosis
- Central nervous system tissue regeneration
SELECTED PUBLICATIONS
- Yang Z, Yao Y, Chen X, Madigan V, Pu S, Fan X, Pu J, Bei F (2025). Cross-species tropism of AAV.CPP.16 in the respiratory tract and its gene therapies against pulmonary fibrosis and viral infection. Cell Reports Medicine DOI: 10.1016/j.xcrm.2025.102144
- Sangster ML, Bishop MM, Yao Y, Feitor JF, Shahriar S, Miller ME, Chekuri AK, Budnik B, Bei F†, Grishchuk Y† (2024). A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice. Molecular Therapy Methods & Clinical Development 32(2):101269. († co-corresponding author)
- Yao Y, Wang J, Liu Y, Qu Y, Wang, K, Zhang Y, Chang Y, Yang Z, Wan J, Liu J, Nakashima H, Lawler SE, Chiocca EA, Cho C, Bei F (2022). Variants of the adeno-associated virus serotype 9 with enhanced penetration of the blood–brain barrier in rodents and primates. Nature Biomedical Engineering. 6(11):1257-1271.
- Norsworthy M*, Bei F*†, Kawaguchi R, Wang C, Wang Q, Li Y, Tran N, Brommer B, Zhang Y, Sanes JR, Coppola G†, He Z† (2017). Sox11 expression promotes regeneration of some retinal ganglion cell types but kills others. Neuron 94(6) 1112-1120. (* co-first author, † co-corresponding author)
- Bei F, Lee HH, Liu X, Gunner G, Jin H, Ma L, Wang C, Hou L, Hensch TK, Frank E, Sanes JR, Chen C, Fagiolini M, He Z (2016). Restoration of visual function by enhancing conduction in regenerated axons. Cell 164(1-2):219-232.