Gene therapy for ultrarare diseases: a geneticist’s perspective DOI Creative Commons
Wuh‐Liang Hwu

Journal of Biomedical Science, Journal Year: 2024, Volume and Issue: 31(1)

Published: Aug. 13, 2024

Abstract Gene therapy has made considerable strides in recent years. More than 4000 protein-coding genes have been implicated more 6000 genetic diseases; next-generation sequencing dramatically revolutionized the diagnosis of diseases. Most diseases are considered very rare or ultrarare, defined here as having fewer 1:100,000 cases, but only one 12 approved gene therapies (excluding RNA therapies) targets an ultrarare disease. This article explores three supplementation approaches suitable for various diseases: lentiviral vector-modified autologous CD34 + hematopoietic stem cell transplantation, systemic delivery adeno-associated virus (AAV) vectors to liver, and local AAV cerebrospinal fluid brain. Together with therapies, we propose a potential business model these therapies.

Language: Английский

Revolutionizing cancer treatment: enhancing CAR-T cell therapy with CRISPR/Cas9 gene editing technology DOI Creative Commons

Ruiyu Tao,

Xiaopeng Han, Xue Bai

et al.

Frontiers in Immunology, Journal Year: 2024, Volume and Issue: 15

Published: Feb. 21, 2024

CAR-T cell therapy, a novel immunotherapy, has made significant breakthroughs in clinical practice, particularly treating B-cell-associated leukemia and lymphoma. However, it still faces challenges such as poor persistence, limited proliferation capacity, high manufacturing costs, suboptimal efficacy. CRISPR/Cas system, an efficient simple method for precise gene editing, offers new possibilities optimizing cells. It can increase the function of cells reduce costs. The combination CRISPR/Cas9 technology therapy may promote development this provide more effective personalized treatment cancer patients. Meanwhile, safety issues surrounding application require further research evaluation. Future should focus on improving accuracy to facilitate better therapy. This review focuses including eliminating inhibitory effect immune checkpoints, enhancing ability resist exhaustion, assisting construction universal cells, reducing costs security problems faced. objective is show revolutionary role researchers.

Language: Английский

Citations

31
Impacts of Climate Change and Mitigation Strategies for Some Abiotic and Biotic Constraints Influencing Fruit Growth and Quality DOI Creative Commons
Eunice Bacelar, Teresa Pinto, Rosário Anjos

et al.

Plants, Journal Year: 2024, Volume and Issue: 13(14), P. 1942 - 1942

Published: July 15, 2024

Factors such as extreme temperatures, light radiation, and nutritional condition influence the physiological, biochemical, molecular processes associated with fruit development its quality. Besides abiotic stresses, biotic constraints can also affect growth Moreover, there be interactions between stressful conditions. However, it is challenging to predict generalize risks of climate change scenarios on seasonal patterns growth, development, yield, quality species because their responses are often highly complex involve changes at multiple levels. Advancements in genetic editing technologies hold great potential for agricultural sector, particularly enhancing crop traits. These improvements tailored meet consumer preferences, which crucial commercial success. Canopy management innovative training systems key factors that contribute maximizing yield efficiency improving quality, essential competitiveness orchards. creation habitats support pollinators a critical aspect sustainable agriculture, they play significant role production many crops, including fruits. Incorporating these strategies allows growers adapt changing conditions, increasingly important stability food production. By investing areas, stay ahead challenges opportunities industry, ultimately leading increased success profitability. In this review, we aim provide an updated overview current knowledge topic. We recommendations future research.

Language: Английский

Citations

27
CRISPR/Cas12a-based biosensors for environmental monitoring and diagnostics DOI Creative Commons
Atif Khurshid Wani, Nahid Akhtar, Tahir ul Gani Mir

et al.

Environmental Technology & Innovation, Journal Year: 2024, Volume and Issue: 34, P. 103625 - 103625

Published: April 4, 2024

Contaminants, such as nucleic acids or toxic small molecules, threaten both human health and ecosystems when they infiltrate the environment. The precise highly sensitive identification of contaminants holds paramount importance across diverse domains, including safeguarding food integrity, facilitating clinical diagnostics, monitoring environmental conditions. Traditional methodologies, encompassing spectroscopy, chromatography, sequencing, metagenomics, have conventionally served pivotal roles in detection processes. Nevertheless, these methods encountered recurring challenges related to sensitivity, specificity, portability. This review focuses on groundbreaking CRISPR/Cas12-based biosensors. These biosensors leverage incredible precision programmability CRISPR/Cas system recognize specific targets. Here, we comprehensively assess fundamental mechanisms that enable detection, ranging from guide RNA design collateral cleavage. versatility CRISPR/Cas12 becomes evident through their applications. applications encompass medical safety, monitoring. transition conventional ultimately represents a significant milestone contaminant detection. By incorporating molecular biology, nanotechnology, bioinformatics, potential reshape landscape water CRIPSR-Cas diagnostics is transformative technology paves way for safer healthier future environment life.

Language: Английский

Citations

21
Actinomycete-Derived Pigments: A Path Toward Sustainable Industrial Colorants DOI Creative Commons
Beatriz Dı́ez, Cristiana A.V. Torres, Susana P. Gaudêncio

et al.

Marine Drugs, Journal Year: 2025, Volume and Issue: 23(1), P. 39 - 39

Published: Jan. 13, 2025

Pigment production has a substantial negative impact on the environment, since mining for natural pigments causes ecosystem degradation, while synthetic pigments, derived from petrochemicals, generate toxic by-products that accumulate and persist in aquatic systems due to their resistance biodegradation. Despite these challenges, remain essential across numerous industries, including cosmetic, textile, food, automotive, paints coatings, plastics, packaging industries. In response growing consumer demand sustainable options, there is increasing interest eco-friendly alternatives, particularly bio-based algae, fungi, actinomycetes. This shift largely driven by options. For bio-pigments, actinomycetes, Streptomyces genus, have emerged as promising green source, aligning with global sustainability goals renewability biodegradability. Scale-up of yield optimization challenges been circumvented aid biotechnology advancements, genetic engineering innovative fermentation extraction methods, which enhanced bio-pigments’ viability cost-competitiveness. Actinomycete-derived successfully transitioned laboratory research commercialization, showcasing potential alternatives dyes. With pigment market valued at approximately USD 24.28 billion 2023, projected reach 36.58 2030, economic actinomycete extensive. review explores environmental advantages role modern industry, regulatory commercialization they face, highlighting importance solutions reduce our reliance conventional pigments. The successful can drive an industry-wide transition environmentally responsible offering benefits human health, safety, sustainability.

Language: Английский

Citations

2
Effective Delivery of CRISPR/dCas9-SAM for Multiplex Gene Activation Based on Mesoporous Silica Nanoparticles for Bladder Cancer Therapy DOI Creative Commons

Jinming Xu,

Jiaju Xu,

Chengfang Sun

et al.

Acta Biomaterialia, Journal Year: 2025, Volume and Issue: unknown

Published: March 1, 2025

Language: Английский

Citations

2
Recent Developments in CRISPR/Cas9 Genome-Editing Technology Related to Plant Disease Resistance and Abiotic Stress Tolerance DOI Creative Commons
İbrahim Erdoğan,

Birsen Cevher-Keskin,

Özlem Bilir

et al.

Biology, Journal Year: 2023, Volume and Issue: 12(7), P. 1037 - 1037

Published: July 22, 2023

The revolutionary CRISPR/Cas9 genome-editing technology has emerged as a powerful tool for plant improvement, offering unprecedented precision and efficiency in making targeted gene modifications. This practical approach to genome editing offers tremendous opportunities crop surpassing the capabilities of conventional breeding techniques. article provides an overview recent advancements challenges associated with application improvement. potential terms developing crops enhanced resistance biotic abiotic stresses is highlighted, examples genes edited confer disease resistance, drought tolerance, salt cold tolerance. Here, we also discuss importance off-target effects efforts made mitigate them, including use shorter single-guide RNAs dual Cas9 nickases. Furthermore, alternative delivery methods, such protein- RNA-based approaches, are explored, they could potentially avoid integration foreign DNA into genome, thus alleviating concerns related genetically modified organisms (GMOs). We emphasize significance accelerating processes, reducing time costs, enabling introduction desired traits at nucleotide level. As field continues evolve, it anticipated that will remain prominent adaptation challenging environmental conditions.

Language: Английский

Citations

34
Emerging frontiers in drug delivery with special focus on novel techniques for targeted therapies DOI Open Access
Ling Yu,

Shengmao Liu,

Shengnan Jia

et al.

Biomedicine & Pharmacotherapy, Journal Year: 2023, Volume and Issue: 165, P. 115049 - 115049

Published: June 25, 2023

The management and treatment of disease are achieved via the use pharmacologically active substances or drugs. Drugs do not, however, have an intrinsic ability to be effective; rather, how well they work depends on administered supplied. Treatment a variety biological illnesses, such as autoimmune disorders, cancer, bacterial infections, requires effective drug delivery. Drug absorption, distribution, metabolism, duration therapeutic impact, pharmacokinetics, excretion, toxicity can all impacted by administration. Improved chemistry materials required for delivery concentration novel treatments specified targets within body, necessary time. This requirement is accompanied development new therapeutics. Formulating medication DDS promising strategy directly addressing numerous typical barriers adherence, frequent dosage, side effects, delayed beginning action. In current review, we give compendium controlled release subsequently highlight some newest developments in realm, with particular emphasis cutting-edge methods targeted therapy. each instance, outline obstacles efficient administration chemical material that allowing sector overcome these positive clinical impact.

Language: Английский

Citations

27
Multidrug-Resistant tuberculosis DOI

Dika Apriliana Wulandari,

Yeni Wahyuni Hartati, Abdullahi Umar Ibrahim

et al.

Clinica Chimica Acta, Journal Year: 2024, Volume and Issue: 559, P. 119701 - 119701

Published: May 1, 2024

Language: Английский

Citations

15
CRISPR/Cas genome editing in plants: mechanisms, applications, and overcoming bottlenecks DOI
Delight Hwarari,

Yasmina Radani,

Yongchao Ke

et al.

Functional & Integrative Genomics, Journal Year: 2024, Volume and Issue: 24(2)

Published: March 5, 2024

Language: Английский

Citations

13
Advances in Nanoparticles as Non-Viral Vectors for Efficient Delivery of CRISPR/Cas9 DOI Creative Commons

Minse Kim,

Youngwoo Hwang,

Seongyu Lim

et al.

Pharmaceutics, Journal Year: 2024, Volume and Issue: 16(9), P. 1197 - 1197

Published: Sept. 11, 2024

The clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 system is a gene-editing technology. Nanoparticle delivery systems have attracted attention because of the limitations conventional viral vectors. In this review, we assess efficiency various nanoparticles, including lipid-based, polymer-based, inorganic, and extracellular vesicle-based systems, as non-viral vectors for CRISPR/Cas9 delivery. We discuss their advantages, limitations, current challenges. By summarizing recent advancements highlighting key strategies, review aims to provide comprehensive overview role in advancing technology clinical applications gene therapy.

Language: Английский

Citations

10