Members

Research Interests: Dr. Young is a research scientist specializing in the field of Genetics and Metabolism. His main focus is molecular principles of auditory information processing and Gene Therapy approaches to treat neurological disorders.

Research interests:Dr. Samulski’s research focuses on the study of the dependent parvovirus adeno-associated virus (AAV).

Research Interests: The efficacy of the lentiviral vector system: Production systems, vector titer, vector specific activity, duration of transgene expression. The safety of the retro/lentiviral vector system: Vector mobilization, insertional mutagenesis, contamination with endogenous retroviruses. Basic HIV biology: The role of episomal HIV in the viral lifecycle, variations of RT products, HIV silencing, packaging signal mapping.

Research Interests: Dr. McCown’s overall research entails the development of viral vector gene therapy for the treatment of neurological disorders. With a primary focus upon intractable temporal lobe epilepsy, Dr. McCown continues to refine the use of site directed gene therapy, as well as advance the potential of an intravenous AAV gene therapy. In addition, Dr. McCown has employed AAV capsid DNA shuffling and directed evolution in order to discover novel AAV vectors that exhibit unique, therapy related properties. These studies have resulted in the discovery of chimeric AAV vectors that upon intravenous administration, can selectively cross the seizure compromised bloodbrain barrier, as well as novel vectors that exhibit unique cell specific tropism in the CNS.

Research Interest: Dr. Li’s core research theme is to explore novel strategies for development of AAV vectors with the ability to target human tissues and evade AAV immunity.

Research Interests: Dr. Hirsch works on Adeno-Associated Virus (AAV) biology and the development AAV Gene Therapeutics with a focus on Synthetic inverted terminal repeats, Ocular therapeutics, and DNA damage response, stemness, differentiation, and cancers.

Research Interests: Dr. Bobo’s research specializes in molecular mechanisms underlying rare congenital neurogenetic disorders and translating these insights into innovative gene therapies, with a primarily focus on lysosomal storage disorders (LSDs), including Mucopolysaccharidosis (MPS), and other severe neurodevelopmental conditions for which there are currently no effective treatments.

Research Interests: Intrinsic AAV proteins and their potential roles in AAV biology that can be applied to AAV gene therapies in the Central Nervous System (CNS).

Research Interests: Dr. Song’s primary focus has been on advancing the development of safer and more effective AAV vectors for liver-targeted gene therapy.

Research Interests: Gene therapy for hemophilia, hemophilia associated joint disease (Hemophilia Arthropathy), by expressing bypassing agents (activated coagulation factor V, IX) via AAV vectors)

Research interests: Dr. Hathaway’s research focuses on unraveling the mechanisms of epigenetic gene regulation by developing chemical biology-based tools that can control chromatin state and consequently gene expression. Our team is currently working on applying these chemical epigenetic modifying technologies to regulate genetic payloads from gene therapeutic vectors. Our ultimate goal is to develop a versatile solution that can use this dose responsive platform effectively address rare monogenic diseases. 

Research Interests: Yevgeny (Yev) Brudno’s lab creates innovative biomaterials that improve the engineering, potency, and delivery of cell therapies such as CAR T cells. His team works at the interface of materials science and molecular medicine to reimagine how advanced therapies are designed and brought to patients.

Research Interests: Alison Axtman’s lab, which is part of SGC-UNC, focuses on the synthesis of openly shared small molecules that selectively modulate or degrade proteins implicated in disease-propagating pathways, especially those impacting the brain. The lab operates at the interface between chemistry and biology.