id	author	title	date	pages	extension	mime	words	sentence	flesch	summary	cache	txt
gs-4427	Sulashvili, Nodar; Seniuk, Igor; Kravchenko, Vira; Imerlishvili, Irina; Gabunia, Luiza; Gorgaslidze, Nana; Buleishvili, Maka; Sulashvili, Marika; Singh, Rajkaran; Gundluru, Deva Harsha Uday; Aphkhazava, David	ADVANCES, CLINICAL TRANSLATION AND EMERGING CHALLENGES IN GENE THERAPY: FROM VIRAL VECTOR ENGINEERING AND GENOME EDITING TO PRECISION AND PERSONALIZED GENETIC MEDICINE	2025	27	.pdf	application/pdf	11016	545	18	Traditional drug development paradigms emphasizing large randomized controlled trials establishing safety and efficacy through comparison to standard of care prove challenging for gene therapies targeting rare diseases where patient populations are small, disease heterogeneity is high, and ethical concerns arise regarding randomization to placebo or conventional therapy when gene therapy shows promise in early studies. PhD, Doctor of Biological Sciences, Invited Professor of Biochemistry of University of Georgia, Tbilisi Georgia; Orcid: https://orcid.org/0000-0001-6216-6477 Abstract The advent of genome editing technologies, most notably CRISPR-Cas systems, has further expanded the therapeutic scope of gene therapy by enabling precise modification of endogenous DNA.	cache/gs-4427.pdf	txt/gs-4427.txt