Synthetic Nucleic Acid Analogues in Gene Therapy: An Update for Peptide–Oligonucleotide Conjugates

Taskova, Maria; Mantsiou, Anna; Astakhova, Kira

doi:10.1002/cbic.201700229

Cited by 30 publications

(19 citation statements)

References 91 publications

(197 reference statements)

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“…Short synthetic oligonucleotides with advantages of high homogeneity, controlled purity and known sequences [ [22] , [23] , [24] , [25] ], have been repeatedly reported to bind a-DNA/RNA antibodies [ 17 , 18 ]. Furthermore, ANAs are frequently associated with different autoimmune diseases including SLE and chronic uveitis [ 8 ].…”

Section: Discussionmentioning

confidence: 99%

Serological comparison of systemic lupus erythematosus with neuropsychiatric lupus using synthetic nucleic acid antigens

Khatri

Psaraftis

Funaro

et al. 2020

Journal of Translational Autoimmunity

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Neuropsychiatric systemic lupus erythematosus is an autoimmune disorder characterized by an irregular exchange between the central nervous system and the immune system, leading to the outbreak of neurological conditions with possible disabling effects. Although neuropsychiatric systemic lupus erythematosus is the most common expression of lupus condition, it is still poorly understood. In this study, we focus on the development of an advantageous method based on the application of synthetic nucleic acids and protein-based antigen arrays in order to characterize autoreactive antibodies in neuropsychiatric systemic lupus erythematosus. We confirmed the benefits of using synthetic oligonucleotides such as assay reproducibility, elevated affinity and specificity to autoreactive antibodies. We also demonstrated presence of autoantibodies towards three particular synthetic double stranded antigens and verify similarity of antinuclear antibody patterns in ordinary lupus and neuropsychiatric systemic lupus erythematosus.

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Section: Discussionmentioning

confidence: 99%

Serological comparison of systemic lupus erythematosus with neuropsychiatric lupus using synthetic nucleic acid antigens

Khatri

Psaraftis

Funaro

et al. 2020

Journal of Translational Autoimmunity

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“…This different outcome might be the result of different availability in serum and a half-life of both forms of oligos, and many other issues associated with technical aspects of gene therapy. Morpholino oligos seem to have many obvious advantages over RNA-based oligos, also chemically modified in cellular model and in mice model for short term studies [147,148,149]. Their advantage might be limited though for long term studies, bigger animal studies and for clinical trials when long term effects are expected and without frequent deliveries.…”

Section: Gene Therapy Strategies Tested So Far For Progeriamentioning

confidence: 99%

Hutchinson-Gilford Progeria Syndrome—Current Status and Prospects for Gene Therapy Treatment

Piekarowicz

Machowska

Dzianisava

et al. 2019

Cells

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Hutchinson-Gilford progeria syndrome (HGPS) is one of the most severe disorders among laminopathies—a heterogeneous group of genetic diseases with a molecular background based on mutations in the LMNA gene and genes coding for interacting proteins. HGPS is characterized by the presence of aging-associated symptoms, including lack of subcutaneous fat, alopecia, swollen veins, growth retardation, age spots, joint contractures, osteoporosis, cardiovascular pathology, and death due to heart attacks and strokes in childhood. LMNA codes for two major, alternatively spliced transcripts, give rise to lamin A and lamin C proteins. Mutations in the LMNA gene alone, depending on the nature and location, may result in the expression of abnormal protein or loss of protein expression and cause at least 11 disease phenotypes, differing in severity and affected tissue. LMNA gene-related HGPS is caused by a single mutation in the LMNA gene in exon 11. The mutation c.1824C > T results in activation of the cryptic donor splice site, which leads to the synthesis of progerin protein lacking 50 amino acids. The accumulation of progerin is the reason for appearance of the phenotype. In this review, we discuss current knowledge on the molecular mechanisms underlying the development of HGPS and provide a critical analysis of current research trends in this field. We also discuss the mouse models available so far, the current status of treatment of the disease, and future prospects for the development of efficient therapies, including gene therapy for HGPS.

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“…The ability to chemically assemble oligodeoxynucleotide (ODN) on a solid support efficiently has made a broad impact in the last few decades on many research areas including molecular biology, [1–4] chemical biology, [4–9] synthetic biology, [9–10] data storage, [11–13] nanotechnology [1,5] and medicine. [1–6] However, the success of ODN synthesis is mainly limited to unmodified ODNs. For modified ODNs, we still face many challenges.…”

Section: Introductionmentioning

confidence: 99%

Incorporation of Sensitive Ester and Chloropurine Groups into Oligodeoxynucleotides through Solid‐Phase Synthesis

et al. 2018

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Nucleosides containing ester groups that are sensitive to nucleophiles were incorporated into oligodeoxynucleotides (ODNs) through solid phase chemical synthesis. The sensitive esters are located on a purine nucleobase. They are the esters of ethyl, 2‐methoxyethyl, 4‐methoxyphenyl and phenyl groups, and a thioester. These esters cannot survive the deprotection and cleavage conditions used in known ODN synthesis technologies, which involve strong nucleophiles such as ammonium hydroxide and potassium methoxide (potassium carbonate in anhydrous methanol). To incorporate these sensitive groups into ODNs, the Dmoc (i. e. dimethyl‐1,3‐dithian‐2‐ylmethoxycarbonyl) phosphoramidites and linker were used for solid phase synthesis, which allowed ODN deprotection and cleavage to be carried out under non‐nucleophilic oxidative conditions. Sixteen ODN sequences containing these groups were synthesized and characterized with MALDI MS. In addition, the synthesis and characterization of three ODNs containing a nucleophile sensitive 6‐chloropurine using the same strategy are described.

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Synthetic Nucleic Acid Analogues in Gene Therapy: An Update for Peptide–Oligonucleotide Conjugates

Cited by 30 publications

References 91 publications

Serological comparison of systemic lupus erythematosus with neuropsychiatric lupus using synthetic nucleic acid antigens

Serological comparison of systemic lupus erythematosus with neuropsychiatric lupus using synthetic nucleic acid antigens

Hutchinson-Gilford Progeria Syndrome—Current Status and Prospects for Gene Therapy Treatment

Incorporation of Sensitive Ester and Chloropurine Groups into Oligodeoxynucleotides through Solid‐Phase Synthesis

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