Literature
Primary literature citations for gene therapy components. Each reference includes full citation details, DOI links, and key findings. These publications establish the foundational knowledge for nuclease engineering, AAV serotype discovery, base editor development, and epigenome editing systems used in modern gene therapy.
| Citation | Journal | Title | Citations |
|---|---|---|---|
| Abudayyeh et al. (2017) | Nature | RNA targeting with CRISPR-Cas13 | 2384 |
| Adra et al. (1987) | Gene | Cloning and expression of the mouse pgk-1 gene and the nucleotide sequence of its promoter | 427 |
| Anastassiadis et al. (2009) | Dis Model Mech | Dre recombinase, like Cre, is a highly efficient site-specific recombinase in E. coli, mammalian cells and mice | 0 |
| Anzalone et al. (2019) | Nature | Search-and-replace genome editing without double-strand breaks or donor DNA | 5334 |
| Atchison et al. (1965) | Science | Adenovirus-associated defective virus particles | 1516 |
| Bantel-Schaal et al. (1999) | J Virol | Human adeno-associated virus type 5 is only distantly related to other known primate helper-dependent parvoviruses | 260 |
| Beyer et al. (2015) | PLoS ONE | AQUA Cloning: A Versatile and Simple Enzyme-Free Cloning Approach | — |
| Boshart et al. (1985) | Cell 41:521-530 | A very strong enhancer is located upstream of an immediate early gene of human cytomegalovirus | 1844 |
| Bowles et al. (2012) | Mol Ther | Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector | 516 |
| Brummelkamp et al. (2002) | Science | A system for stable expression of short interfering RNAs in mammalian cells | 6921 |
| Buck et al. (2020) | Int J Mol Sci | Recombinant adeno-associated viral vectors (rAAV)-vector elements in ocular gene therapy clinical trials and transgene expression and bioactivity assays | 0 |
| Campeau et al. (2009) | PLoS ONE | Campeau et al. 2009 | — |
| Carroll et al. (2016) | Proc Natl Acad Sci USA | A mouse model for adult cardiac-specific gene deletion with CRISPR/Cas9 | 238 |
| Cearley et al. (2008) | Mol Ther | Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain | 236 |
| Chalfie et al. (1994) | Science | Green fluorescent protein as a marker for gene expression | 10788 |
| Challis et al. (2019) | Nat Protoc | Systemic AAV vectors for widespread and targeted gene delivery in rodents | 470 |
| Chan et al. (2017) | Nat Neurosci | Engineered AAVs for efficient noninvasive gene delivery to the central nervous system | 1674 |
| Chapman et al. (1991) | Nucleic Acids Res. | Effect of intron A from human cytomegalovirus (Towne) immediate-early gene on heterologous expression in mammalian cells | — |
| Chavez et al. (2015) | Nat Methods | Highly efficient Cas9-mediated transcriptional programming | 1940 |
| Chen et al. (2021) | Nat Commun | Programmable C:G to G:C genome editing with CRISPR-Cas9-directed base excision repair proteins | 218 |
| Chen et al. (2021) | Cell | Enhanced prime editing systems by manipulating cellular determinants of editing outcomes | 811 |
| Chiu et al. (1996) | Curr Biol | Engineered GFP as a vital reporter in plants | 0 |
| Choudhury et al. (2016) | Mol Ther | Viral vectors for therapy of neurologic diseases | 240 |
| Cong et al. (2013) | Science | Multiplex genome engineering using CRISPR/Cas systems | 20340 |
| Conti et al. (1996) | Structure | Crystal structure of firefly luciferase throws light on a superfamily of adenylate-forming enzymes | 0 |
| Cormack et al. (1996) | Gene | FACS-optimized mutants of the green fluorescent protein (GFP) | 4491 |
| Daniels et al. (2014) | PLoS ONE | Expression of Multiple Transgenes from a Single Construct Using Viral 2A Peptides in Drosophila | — |
| Davis et al. (2022) | Nat Biomed Eng | Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors | 192 |
| Deken et al. (2016) | Int J Mol Sci | Dermal delivery of constructs encoding Cre recombinase to induce skin tumors in PtenLoxP/LoxP;BrafCA/+ mice | 0 |
| DeLuca et al. (1978) | Methods Enzymol | Purification and properties of firefly luciferase | 0 |
| DeNoto et al. (1981) | Nucleic Acids Res | Human growth hormone DNA sequence and mRNA structure: possible alternative splicing | 650 |
| Deverman et al. (2016) | Nat Biotechnol | Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain | 1277 |
| Deverman et al. (2018) | Nat Protoc | Gene therapy for neurological disorders: progress and prospects | 430 |
| Diao & White (2011) | Genetics | A Novel Approach for Directing Transgene Expression in Drosophila: T2A-Gal4 In-Frame Fusion | — |
| Edraki et al. (2019) | Mol Cell | A compact, high-accuracy Cas9 with a dinucleotide PAM for in vivo genome editing | 322 |
| Esvelt et al. (2013) | Nat Methods | Orthogonal Cas9 proteins for RNA-guided gene regulation and editing | 1172 |
| Evan et al. (1985) | Mol Cell Biol | Isolation of monoclonal antibodies specific for human c-myc proto-oncogene product | 3200 |
| Excoffon et al. (2009) | Proc Natl Acad Sci USA | Directed evolution of adeno-associated virus to an infectious respiratory virus | 241 |
| Foust et al. (2009) | Nat Biotechnol | Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes | 1754 |
| Fraser et al. (1996) | Insect Molecular Biology | Precise excision of TTAA-specific lepidopteran transposons piggyBac (IFP2) and tagalong (TFP3) from the baculovirus genome in cell lines from two species of Lepidoptera | 0 |
| Gao et al. (2002) | Proc Natl Acad Sci USA | Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy | 2065 |
| Gao et al. (2004) | J Virol | Clades of Adeno-associated viruses are widely disseminated in human tissues | 1509 |
| Gaudelli NM, Komor AC, Rees HA, et al. (2017) | Nature 551:464-471 | Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage | 4827 |
| Gaudelli et al. (2020) | Nat Biotechnol | Directed evolution of adenine base editors with increased activity and therapeutic application | 665 |
| George et al. (2017) | N Engl J Med | Hemophilia B gene therapy with a high-specific-activity factor IX variant | 887 |
| Goedhart et al. (2012) | Nature Communications | Structure-guided evolution of cyan fluorescent proteins towards a quantum yield of 93% | — |
| Goodwin & Rottman (1992) | J Biol Chem | The 3'-flanking sequence of the bovine growth hormone gene contains novel elements required for efficient and accurate polyadenylation | 162 |
| Gossen et al. (1992) | Proc Natl Acad Sci USA | Tight control of gene expression in mammalian cells by tetracycline-responsive promoters | 7281 |
| Gossen et al. (1995) | Science | Transcriptional activation by tetracyclines in mammalian cells | 3720 |
| Gradinaru et al. (2008) | Brain Cell Biol | eNpHR: a Natronomonas halorhodopsin enhanced for optogenetic applications | 0 |