Global Perspectives on Pharmacogenomics and Drug Discovery DOI
K. Rajakumari,

K. Kavinaya Shri,

R. Logesh

и другие.

Advances in medical technologies and clinical practice book series, Год журнала: 2024, Номер unknown, С. 123 - 166

Опубликована: Дек. 13, 2024

Pharmacogenomics, the study of how genes influence an individual's response to drugs, is changing scene medication disclosure and improvement by joining pharmacology genomics make powerful, custom-made hereditary profile. This arising field vital in customized medication, where medicines are upgraded light individual varieties. chapter offers in-depth exploration global impact pharmacogenomics, underscoring key examination foundations undertakings, moral contemplations, difficulties future headings. The joint efforts exhibit capability endeavor defeat advance fitting worldwide guidelines creating strategies that help even-handed admittance pharmacogenomic progressions significant for reconciliation medication.

Язык: Английский

From bench to bedside: cutting-edge applications of base editing and prime editing in precision medicine DOI Creative Commons

Weihui Xu,

Shiyao Zhang, Huan Qin

и другие.

Journal of Translational Medicine, Год журнала: 2024, Номер 22(1)

Опубликована: Дек. 20, 2024

Abstract CRISPR-based gene editing technology theoretically allows for precise manipulation of any genetic target within living cells, achieving the desired sequence modifications. This revolutionary advancement has fundamentally transformed field biomedicine, offering immense clinical potential treating and correcting disorders. In treatment most diseases, genome that avoids generation mixed byproducts is considered ideal approach. article reviews current progress base editors prime editors, elaborating on specific examples their applications in therapeutic field, highlights opportunities improvement. Furthermore, we discuss performance these technologies terms safety efficacy applications, analyze latest advancements directions could influence future development technologies. Our goal to outline relevance this rapidly evolving scientific preview a roadmap successful DNA therapies hereditary or idiopathic diseases.

Язык: Английский

Процитировано

5
The Applications of Artificial Intelligence (AI)-Driven Tools in Virus-Like Particles (VLPs) Research DOI

Bugude Laxmi,

P. Uma Maheswari Devi,

Naveen Thanjavur

и другие.

Current Microbiology, Год журнала: 2024, Номер 81(8)

Опубликована: Июнь 21, 2024

Язык: Английский

Процитировано

4
The Current State of Cytotherapy and the Field of Cell and Gene Therapy DOI Creative Commons
William Ying Khee Hwang,

Ezzah Mohamed Muzammil

Cytotherapy, Год журнала: 2025, Номер unknown

Опубликована: Фев. 1, 2025

Язык: Английский

Процитировано

0
Efficient solid-phase extraction of oligo-DNA from complex media using a nitrocellulose membrane modified with carbon nanotubes and aminated reduced graphene oxide DOI Creative Commons

Georgian Alin Toader,

Dumitru Mihalache, Valentin Titus Grigorean

и другие.

Scientific Reports, Год журнала: 2025, Номер 15(1)

Опубликована: Фев. 13, 2025

Язык: Английский

Процитировано

0
The Role of Non-coding RNAs in Diabetic Retinopathy: Mechanistic Insights and Therapeutic Potential DOI

Zhuan Zuo,

Ni Li, Qian Zhang

и другие.

Molecular Neurobiology, Год журнала: 2025, Номер unknown

Опубликована: Апрель 1, 2025

Язык: Английский

Процитировано

0
Anesthetic effects on electrophysiological responses across the visual pathway DOI Creative Commons
Shiyao Zhang,

Weihui Xu,

Shanshan Liu

и другие.

Scientific Reports, Год журнала: 2024, Номер 14(1)

Опубликована: Ноя. 13, 2024

Anesthetics are widely used in electrophysiological tests to assess retinal and visual system functions avoid experimental errors caused by movement stress animals. To determine the most suitable anesthetic for tests, excluding ketamine chloral hydrate due regulatory side effect concerns, this study investigated effects of ethyl carbamate (EC), avertin (AR), pentobarbital sodium (PS) on signal conduction retina primary cortex. Assessments included flash electroretinogram (FERG), pattern (PERG), evoked potentials (PVEP), (FVEP), FERG FVEP were evaluate responses cortex stimuli, respectively, while PERG PVEP assessed stimuli. The research showed that AR demonstrates least disruption pathway, as evidenced consistently high characteristic peaks group across various tests. In contrast, mice given EC exhibited lowest peak values both FVEP, subjects anesthetized with PS suppressed oscillatory responses. Notably, substantial observed only AR. Consequently, among three anesthetics tested, is studies.

Язык: Английский

Процитировано

1
Base editing in humanized dystrophic mice DOI Creative Commons
Chen Zhang, Yuan Zhou, Renzhi Han

и другие.

Molecular Therapy — Nucleic Acids, Год журнала: 2024, Номер 35(2), С. 102185 - 102185

Опубликована: Апрель 12, 2024

In vivo gene editing and, more recently, base have shown great promise in correcting the dystrophin mutations animal models and patient cells. A recent study published Molecular Therapy Nucleic Acids demonstrates efficient correction of nonsense human humanized mouse Duchenne muscular dystrophy (DMD) by adenine (ABE).1Jin M. Lin J. Li H. Z. Yang D. Wang Y. Yu Shao Chen L. et al.Correction mutation via for treatment mouse.Mol. Ther. Acids. 2024; 35102165https://doi.org/10.1016/j.omtn.2024.102165Google Scholar DMD is characterized absence functional protein resulting from gene, leading to progressive muscle wasting, cardiac complications, premature mortality. Large deletion account approximately 70% all cases. The major therapeutic efforts are centered on restoring expression. One strategy deliver a miniaturized version cDNA, known as micro-dystrophin, through adeno-associated virus (AAV). 2023, US Food Drug Administration (FDA) granted accelerated approval Sarepta's Elevidys (AAVrh.74 carrying micro-dystrophin cDNA) patients with who 4–5 years age.2Hoy S.M. Delandistrogene Moxeparvovec: First Approval.Drugs. 2023; 83: 1323-1329https://doi.org/10.1007/s40265-023-01929-xGoogle This represents an important milestone therapy development. Another utilizes antisense oligonucleotides induce targeted exon skipping restore reading frame gene. Exon converts DMD-associated frame-disrupting into Becker dystrophy-like deletions, which can produce functional, truncated protein. first exon-skipping drug 51 skipping, eteplirsen, received FDA 2016, followed VyonDys-53 viltolarsen 53 AmonDys-45 45 skipping. More CRISPR-Cas9 system has permanently demonstrated numerous preclinical studies cell culture studies.3Roberts T.C. Wood M.J.A. Davies K.E. Therapeutic approaches dystrophy.Nat. Rev. Discov. 22: 917-934https://doi.org/10.1038/s41573-023-00775-6Google While large deletions most common cause DMD, estimated 10%–15% Correcting such could take different strategies, potential full-length involves modulating translation termination efficiency utilization pharmacological agents or engineered suppressor tRNAs.4Ng M.Y. Ghelfi M.D. Goldman Y.E. Cooperman B.S. Ataluren aminoglycosides stimulate read-through codons orthogonal mechanisms.Proc. Natl. Acad. Sci. USA. 2021; 118e2020599118https://doi.org/10.1073/pnas.2020599118Google Scholar,5Albers S. Allen E.C. Bharti N. Davyt Joshi Perez-Garcia C.G. Santos Mukthavaram R. Delgado-Toscano M.A. Molina B. al.Engineered tRNAs suppress cells vivo.Nature. 618: 842-848https://doi.org/10.1038/s41586-023-06133-1Google Ataluren, oxadiazole compound facilitating ribosomal readthrough stop codons, was approved treat European several other countries. highly promising approach leverages base-editing technology, particular ABE, reverse mutations, offering solution wild-type, expression point mutations. previously been DMD.6Xu Zhang C. P. Gao Mokadam N.A. Ma Arnold W.D. Han Efficient precise adult dystrophic mice.Nat. Commun. 12: 3719https://doi.org/10.1038/s41467-021-23996-yGoogle Scholar,7Ryu Koo T. Kim K. Lim Baek G. S.T. H.S. D.E. Lee Chung E. J.S. Adenine embryos model Biotechnol. 2018; 36: 536-539https://doi.org/10.1038/nbt.4148Google As sequence-context dependent, Jin al. further explored this patient-derived mutations.1Jin authors identified 12 cohort 27 DMD. By screening panel single guide RNAs (sgRNAs) targeting these using fluorescence reporter assay HEK293T cells, they investigated feasibility SpG-ABE. SpG-ABE-mediated varied across reaching up 80% some being marginal others. ∼60% confirmed induced pluripotent stem (iPSC) that generated c.4174C>T 30. After differentiating corrected iPSC colonies cardiomyocytes, restored level comparable normal cardiomyocytes derived H9 immunofluorescence staining. Deep sequencing analysis revealed minimal bystander off-target events, demonstrating selected sgRNA combined SpG-ABE be specific correct To assess performance SpG-ABE/sgRNA, two models, each harboring split intein packaged AAV9 vectors, commonly used capsid delivering genes skeletal muscles. Both ubiquitous muscle-specific promoters were tested drive ABE expression, former exhibiting superior undifferentiated iPSCs latter performing better animals, suggesting selection affect outcomes. Intraperitoneal delivery vectors components resulted widespread rescue treated animals. also reduced serum creatine kinase levels improved function mice, evidenced rotarod treadmill running tests. Muscle fibrosis treatment, histological analysis. comparison ataluren act at transcript level, directly corrects disease-causing chromosomal DNA potentially robust long-lasting benefits. addition, permanent splicing sites enhancers.8Wang Zhu R.Y. Guo Correction iPSC-derived base-editing-induced skipping.Mol. Methods Clin. Dev. 28: 40-50https://doi.org/10.1016/j.omtm.2022.11.010Google Scholar,9Qiu Yuan F. Dai Chang X. base-editor-mediated abrogation exonic enhancers.Cell Rep. 42113340https://doi.org/10.1016/j.celrep.2023.113340Google Besides widely pursued target many genetic even non-genetic diseases, including hearing loss, blindness, immunodeficiency, cardiovascular blood disorders, heart failure, liver neurodegenerative viral infection, among others.10Xu Zheng Xu W. Liu Yao Breaking shackles: advance disorder treatment.Front. Pharmacol. 151364135https://doi.org/10.3389/fphar.2024.1364135Google Excitingly, clinical trial involving nonviral PCSK9 initial results heterozygous familial hypercholesterolemia.11Han shows promise.Mol. 32: 1-2https://doi.org/10.1016/j.ymthe.2023.12.001Google Overall, findings presented al.1Jin together previous ongoing offer compelling evidence diseases. However, investigations warranted address challenges associated editing, AAV efficiency/specificity, effects, host immune responses, long-term safety. Nevertheless, rapid advancement instills hope families affected currently incurable R.H. supported National Institutes Health grants (R01HL169976, R01HL116546, R01HL159900, R01HL170260, R21HL163720). associate editor editorial board member

Язык: Английский

Процитировано

0
Perspectives on CRISPR Genome Editing to Prevent Prion Diseases in High-Risk Individuals DOI Creative Commons

Milan M. Medd,

Qi Cao

Biomedicines, Год журнала: 2024, Номер 12(8), С. 1725 - 1725

Опубликована: Авг. 1, 2024

Prion diseases are neurodegenerative disorders caused by misfolded prion proteins. Although rare, the said always fatal; they commonly cause death within months of developing clinical symptoms, and their diagnosis is exceptionally difficult pre-mortem. There no known cures or treatments other than symptomatic care. Given aggressiveness on onset, therapies after disease onset could be challenging. Prevention to reduce incidence delay has been suggested a more feasible approach. In this perspective article, we summarize our current understandings origin, risk factors, manifestations diseases. We propose PCR testing blood identify

Язык: Английский

Процитировано

0
Global Perspectives on Pharmacogenomics and Drug Discovery DOI
K. Rajakumari,

K. Kavinaya Shri,

R. Logesh

и другие.

Advances in medical technologies and clinical practice book series, Год журнала: 2024, Номер unknown, С. 123 - 166

Опубликована: Дек. 13, 2024

Pharmacogenomics, the study of how genes influence an individual's response to drugs, is changing scene medication disclosure and improvement by joining pharmacology genomics make powerful, custom-made hereditary profile. This arising field vital in customized medication, where medicines are upgraded light individual varieties. chapter offers in-depth exploration global impact pharmacogenomics, underscoring key examination foundations undertakings, moral contemplations, difficulties future headings. The joint efforts exhibit capability endeavor defeat advance fitting worldwide guidelines creating strategies that help even-handed admittance pharmacogenomic progressions significant for reconciliation medication.

Язык: Английский

Процитировано

0