TABLE 4.

Nucleic acids in clinical trials for AD.

Nucleic acid	Vector	Trial start date (end date)	Therapeutic goal	Type of trial (status)	Trial ID	Primary outcome measures	References
BDNF	adeno-associated virus (AAV) vector	7 February 2022	Reduction of neuronal loss and rebuilding synapses in the brain of patients with Cognitive Impairment and AD	Open-label Phase 1 trial	NCT05040217	a. The number of individuals with adverse events related to therapy, evaluated on MRI scan	Tuszynski (2022)
		(1 October 2027)		(Recruiting)		b. Assessment of memory via Ray Auditory Verbal Learning Task
						c. Assessment of Memory with Benson Complex Figure Draw and Memory
APOE2	adeno-associated virus (AAV) vector	6 November 2019	Maximum tolerable dose, and conversion of APOE protein variants in the CSF of individuals with APOE4 homozygosity into APOE2-APOE4	Open-label Phase 1 trial	NCT03634007	a. Proportion of participants reporting serious adverse events and treatment-related adverse events, 12 months post-trial initiation	Lexeo (2019)
		(November 2024)		(Active, not recruiting)		b. Proportion of study reporting adverse events associated with treatment at each dosage