The future of complement therapeutics DOI Creative Commons
Martin Kolev,

Kollu N. Rao,

Michael W. Yeh

и другие.

Exploration of Immunology, Год журнала: 2024, Номер unknown, С. 577 - 615

Опубликована: Окт. 18, 2024

Complement is both evolutionary and scientifically old. It predates the adaptive immunity by some 600 million years was first described in 1905 Jules Bordet Paul Ehrlich. For most of its, existence complement system has been ignored scientists clinicians due to perception it being complicated its relevance for pathogenesis human disease unclear. With recent US Food Drug Administration (FDA) approvals pegcetacoplan paroxysmal nocturnal haemoglobinuria (PNH) geographic atrophy (GA), avacincaptad pegol GA iptacopan danicopan PNH, we are at a crucial juncture complement-targeting therapies. A number companies academic institutions developing next-generation therapies, which resulting an increasingly competitive landscape. If one looks serum cascade, all 3 pathways now have biotechnology or pharmaceutical industry players with 1 multiple clinical-stage inhibitors that expected be FDA approved within next few years. Furthermore, limited clinically validated targets complement-mediated disease, competition this space set further intensify coming In review, will discuss timeline discoveries led development current crop FDA-approved therapeutics. We follow discussion crowded therapy scientific advances emerged two decades underpinning future innovation, including our understanding biology, such as local intracellular complement, emerging targets, combinational approaches non-complement therapeutics unlock new indications technologies gene therapy. also give comprehensive overview landscape how can utilized target dysregulation.

Язык: Английский

Comprehensive Overview of Bottom-Up Proteomics Using Mass Spectrometry DOI Creative Commons
Yuming Jiang, Rex Devasahayam Arokia Balaya, Dina Schuster

и другие.

ACS Measurement Science Au, Год журнала: 2024, Номер 4(4), С. 338 - 417

Опубликована: Июнь 4, 2024

Proteomics is the large scale study of protein structure and function from biological systems through identification quantification."Shotgun proteomics" or "bottom-up prevailing strategy, in which proteins are hydrolyzed into peptides that analyzed by mass spectrometry.Proteomics studies can be applied to diverse ranging simple proteoforms, protein-protein interactions, structural alterations, absolute relative quantification, post-translational modifications, stability.To enable this range different experiments, there strategies for proteome analysis.The nuances how proteomic workflows differ may challenging understand new practitioners.Here, we provide a comprehensive overview proteomics methods.We cover biochemistry basics extraction interpretation orthogonal validation.We expect Review will serve as handbook researchers who field bottom-up proteomics.

Язык: Английский

Процитировано

30
Recent advances of CRISPR-based genome editing for enhancing staple crops DOI Creative Commons
Feng Chen, Chen D. Lu, Yan Zhao

и другие.

Frontiers in Plant Science, Год журнала: 2024, Номер 15

Опубликована: Сен. 23, 2024

An increasing population, climate change, and diminishing natural resources present severe threats to global food security, with traditional breeding genetic engineering methods often falling short in addressing these rapidly evolving challenges. CRISPR/Cas systems have emerged as revolutionary tools for precise modifications crops, offering significant advancements resilience, yield, nutritional value, particularly staple crops like rice maize. This review highlights the transformative potential of technology, emphasizing recent innovations such prime base editing, development novel CRISPR-associated proteins, which significantly improved specificity, efficiency, scope genome editing agriculture. These enable targeted that enhance tolerance abiotic stresses well biotic stresses. Additionally, plays a crucial role improving crop yield quality by enhancing photosynthetic nutrient uptake, resistance lodging, while also taste, texture, shelf life, content through biofortification. Despite challenges off-target effects, need more efficient delivery methods, ethical regulatory concerns, underscores importance security sustainability It calls continued research integration CRISPR other emerging technologies nanotechnology, synthetic biology, machine learning fully realize its developing resilient, productive, sustainable agricultural systems.

Язык: Английский

Процитировано

19
Enhancing precision in cancer treatment: the role of gene therapy and immune modulation in oncology DOI Creative Commons

Emile M. Youssef,

Brandon Fletcher,

Dannelle Palmer

и другие.

Frontiers in Medicine, Год журнала: 2025, Номер 11

Опубликована: Янв. 13, 2025

Gene therapy has long been a cornerstone in the treatment of rare diseases and genetic disorders, offering targeted solutions to conditions once considered untreatable. As field advances, its transformative potential is now expanding into oncology, where personalized therapies address immune-related complexities cancer. This review highlights innovative therapeutic strategies, including gene replacement, silencing, oncolytic virotherapy, CAR-T cell therapy, CRISPR-Cas9 editing, with focus on their application both hematologic malignancies solid tumors. CRISPR-Cas9, revolutionary tool precision medicine, enables precise editing cancer-driving mutations, enhancing immune responses disrupting tumor growth mechanisms. Additionally, emerging approaches target ferroptosis—a regulated, iron-dependent form death—offering new possibilities for selectively inducing death resistant cancers. Despite significant breakthroughs, challenges such as heterogeneity, evasion, immunosuppressive microenvironment (TME) remain. To overcome these barriers, novel like dual-targeting, armored cells, combination checkpoint inhibitors ferroptosis inducers are being explored. rise allogeneic “off-the-shelf” offers scalable more accessible options. The regulatory landscape evolving accommodate advancements, frameworks RMAT (Regenerative Medicine Advanced Therapy) U.S. ATMP (Advanced Therapy Medicinal Products) Europe fast-tracking approval therapies. However, ethical considerations surrounding CRISPR-based editing—such off-target effects, germline ensuring equitable access—remain at forefront, requiring ongoing oversight. Advances non-viral delivery systems, lipid nanoparticles (LNPs) exosomes, improving safety efficacy By integrating innovations addressing concerns, poised revolutionize cancer treatment, providing durable, effective,

Язык: Английский

Процитировано

4
In vivo vectorization and delivery systems for gene therapies and RNA-based therapeutics in oncology DOI

Julie Schock Vaiani,

Mans Broekgaarden, Jean‐Luc Coll

и другие.

Nanoscale, Год журнала: 2025, Номер unknown

Опубликована: Янв. 1, 2025

Gene and RNA-based therapeutics represent a promising frontier in oncology, enabling targeted modulation of tumor-associated genes proteins. This review explores the latest advances payload vectorization delivery systems developed for vivo cancer treatments. We discuss viral non-viral organic particles, including lipid based nanoparticles polymeric structures, effective transport plasmids, siRNA, self-amplifying RNA therapeutics. Their physicochemical properties, strategies to overcome intracellular barriers, innovations cell-based carriers engineered extracellular vesicles are highlighted. Moreover, we consider oncolytic viruses, novel capsid modifications, approaches that refine tumor targeting immunomodulation. Ongoing clinical trials regulatory frameworks guide future directions emphasize need safe, scalable production. The potential convergence these with combination therapies paves way toward personalized medicine.

Язык: Английский

Процитировано

2
Important applications of DNA nanotechnology combined with CRISPR/Cas systems in biotechnology DOI Creative Commons
Yuqi Huang, Zhongping Chen,

Huacui Huang

и другие.

RSC Advances, Год журнала: 2025, Номер 15(8), С. 6208 - 6230

Опубликована: Янв. 1, 2025

Schematic illustration of several synthetic forms 2D and 3D DNA nanotechnology combined with CRISPR/Cas systems in biotechnology.

Язык: Английский

Процитировано

1
Strategies and Methods for Improving the Efficiency of CRISPR/Cas9 Gene Editing in Plant Molecular Breeding DOI Creative Commons
Junming Zhou,

Xinchao Luan,

Yixuan Liu

и другие.

Plants, Год журнала: 2023, Номер 12(7), С. 1478 - 1478

Опубликована: Март 28, 2023

Following recent developments and refinement, CRISPR-Cas9 gene-editing technology has become increasingly mature is being widely used for crop improvement. The application of CRISPR/Cas9 enables the generation transgene-free genome-edited plants in a short period advantages simplicity, high efficiency, specificity, low production costs, which greatly facilitate study gene functions. In plant molecular breeding, efficiency system proven to be key step influencing effectiveness with improvements recently becoming focus reported scientific research. This review details strategies methods improving editing including Cas9 variant enzyme engineering, effect multiple promoter driven Cas9, gRNA efficient optimization expression strategies. It also briefly introduces CRISPR/Cas12a BE PE precision editing. These are beneficial further development systems field breeding.

Язык: Английский

Процитировано

19
Genome editing based trait improvement in crops: current perspective, challenges and opportunities DOI
Surender Singh,

Roni Chaudhary,

Vadthya Lokya

и другие.

The Nucleus, Год журнала: 2024, Номер 67(1), С. 97 - 126

Опубликована: Март 5, 2024

Язык: Английский

Процитировано

7
Unveiling the Genetic Symphony: Harnessing CRISPR-Cas Genome Editing for Effective Insect Pest Management DOI Creative Commons

J. Komal,

H. R. Desai,

Ipsita Samal

и другие.

Plants, Год журнала: 2023, Номер 12(23), С. 3961 - 3961

Опубликована: Ноя. 24, 2023

Phytophagous insects pose a significant threat to global crop yield and food security. The need for increased agricultural output while reducing dependence on harmful synthetic insecticides necessitates the implementation of innovative methods. utilization CRISPR-Cas (Clustered regularly interspaced short palindromic repeats) technology develop insect pest-resistant plants is believed be highly effective approach in production expenses enhancing profitability farms. Insect genome research provides vital insights into gene functions, allowing better knowledge biology, adaptability, development targeted pest management disease prevention measures. editing technique has capability modify DNA insects, either trigger drive or overcome their resistance specific insecticides. advancements CRISPR its various applications have shown potential developing insect-resistant varieties other strategies through sustainable approach. This could consequences ensuring involves using create modified plants. article critically analyzed discussed challenges associated with exploring utilizing pressure

Язык: Английский

Процитировано

15
Liposome-Based Carriers for CRISPR Genome Editing DOI Open Access
Xing Yin, Romain Harmancey,

David D. McPherson

и другие.

International Journal of Molecular Sciences, Год журнала: 2023, Номер 24(16), С. 12844 - 12844

Опубликована: Авг. 16, 2023

The CRISPR-based genome editing technology, known as clustered regularly interspaced short palindromic repeats (CRISPR), has sparked renewed interest in gene therapy. This is accompanied by the development of single-guide RNAs (sgRNAs), which enable introduction desired genetic modifications at targeted site when used alongside CRISPR components. However, efficient delivery CRISPR/Cas remains a challenge. Successful relies on strategy that can effectively deliver cargo to target site. To overcome this obstacle, researchers have extensively explored non-viral, viral, and physical methods for CRISPR/Cas9 guide RNA (gRNA) into cells tissues. Among those methods, liposomes offer promising approach enhance gRNA. Liposomes facilitate endosomal escape leverage various stimuli such light, pH, ultrasound, environmental cues provide both spatial temporal control release. Thus, combination system with liposome technology enables precise numerous applications basic research, biotechnology, therapeutic interventions. For instance, it be employed correct mutations associated inherited diseases other disorders or modify immune their disease-fighting capabilities. In summary, liposome-based provides valuable tool achieving modifications. review discusses future directions opportunities further advance rapidly evolving field.

Язык: Английский

Процитировано

14
Targeted genome editing for cotton improvement: prospects and challenges DOI
Rakesh Kumar, Joy Das,

Raghavendra Keelara Puttaswamy

и другие.

The Nucleus, Год журнала: 2024, Номер 67(1), С. 181 - 203

Опубликована: Март 21, 2024

Язык: Английский

Процитировано

6