Leveraging Vector-Based Gene Disruptions to Enhance CAR T-Cell Effectiveness DOI Open Access
Beatriz Coutinho de Oliveira, S. M. Nayeemul Bari, J. Joseph Melenhorst

et al.

Cancers, Journal Year: 2025, Volume and Issue: 17(3), P. 383 - 383

Published: Jan. 24, 2025

Anti-CD19 chimeric antigen receptor (CAR) T-cell therapy represents a breakthrough in the treatment of relapsed and refractory B-cell malignancies, such as chronic lymphocytic leukemia (CLL), inducing long-term, sometimes curative, responses. However, fewer than 30% CLL patients achieve outcomes. It has been shown that smaller subset T cells capable expansion persistence is crucial for effectiveness. Notably, pre-existing mutation epigenetic regulator TET2, combined with CAR vector-induced disruption other intact allele, significantly enhanced potency CAR-engineered clone one patient. This finding aligns independent research, suggesting gene’s genomic insertion site influences tumor-targeting capability. Thus, it plausible gene disruptions affect function. review synthesizes existing knowledge on vector integration into host genome its impact clinical outcomes patients. Our aim to inform development improved therapies enhance their overall efficacy.

Language: Английский

Mesenchymal stem cells and mesenchymal stem cell-derived exosomes: a promising strategy for treating retinal degenerative diseases DOI Creative Commons

Wenjing An,

Wenliang Zhang, Qi Jia

et al.

Molecular Medicine, Journal Year: 2025, Volume and Issue: 31(1)

Published: Feb. 21, 2025

Abstract Mesenchymal stem cells (MSCs) have emerged as a promising therapeutic strategy in regenerative medicine, demonstrating significant potential for clinical applications. Evidence suggests that MSCs not only exhibit multipotent differentiation but also exert critical effects retinal degenerative diseases via robust paracrine mechanisms. protect from damage by modulating inflammation, inhibiting apoptosis, alleviating oxidative stress, and suppressing cell death pathways. Furthermore, contribute to structural functional stability facilitating vascular remodeling donating mitochondria cells. Of particular interest, MSC-derived exosomes gained widespread attention compelling cell-free therapy. Owing their potent anti-inflammatory, anti-apoptotic, vascular-stabilizing properties, show promise the treatment of diseases.

Language: Английский

Citations

1
Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence DOI Creative Commons

Jimi Olaghere,

D. A. Williams, Jeremy Farrar

et al.

Biomedicines, Journal Year: 2025, Volume and Issue: 13(3), P. 758 - 758

Published: March 20, 2025

On 4 September 2024, the Reagan-Udall Foundation for FDA (FDA Foundation) in collaboration with Food and Drug Administration (FDA) Gates hosted a workshop titled “Scientific Advancements Gene Therapies: Opportunities Global Regulatory Convergence”. The event brought together diverse group of experts, including international regulatory bodies, regulated industries, healthcare professionals, patients, academic researchers global health advocates, to discuss rapid advancements gene therapy pressing need equitable access low-and middle-income countries (LMICs), sickle cell disease (SCD) serving as model disorder discussions. Although there has been significant progress therapy, such breakthroughs clustered regularly interspaced short palindromic repeats (CRISPR)-based technologies FDA-approved therapies, these therapies remain limited underresourced regions. addressed critical challenges, high cost gaps barriers ethical concerns regarding informed consent public engagement LMICs. This paper highlights discussion points from focus on exploring strategies convergence, role collaborations potential pathways making affordable accessible all.

Language: Английский

Citations

0
Advancements in age-related macular degeneration treatment: From traditional anti-VEGF to emerging therapies in gene, stem cell, and nanotechnology DOI
Zhanfei Wang, Yaqin Zhang, Chunxiu Xu

et al.

Biochemical Pharmacology, Journal Year: 2025, Volume and Issue: unknown, P. 116902 - 116902

Published: March 1, 2025

Language: Английский

Citations

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

Zhuan Zuo,

Ni Li, Qian Zhang

et al.

Molecular Neurobiology, Journal Year: 2025, Volume and Issue: unknown

Published: April 1, 2025

Language: Английский

Citations

0
Cell lineage tracing: Methods, applications, and challenges DOI Open Access
Shanjun Mao, Chenyang Zhang,

Runjiu Chen

et al.

Quantitative Biology, Journal Year: 2025, Volume and Issue: 13(4)

Published: May 8, 2025

Abstract Cell lineage tracing is a crucial technique for understanding cell fate and relationships, with wide applications in developmental biology, tissue regeneration, disease progression studies. Over the years, experimental methods have advanced from early labeling techniques to modern genetic tools such as CRISPR‐Cas9‐based barcoding, whereas computational emerged analyze high‐dimensional data single‐cell sequencing other omics technologies. This paper reviews both methods, highlighting their respective strengths, limitations, synergies. Experimental focus on tracking cells, approaches reconstruct relationships model cellular dynamics. Despite significant progress, challenges remain, including issues accuracy, resolution, multi‐omics integration, scalability. Future directions involve improvements development of enhanced by advancements artificial intelligence. These innovations are expected drive field forward, offering potential uncovering mysteries life.

Language: Английский

Citations

0
Leveraging Vector-Based Gene Disruptions to Enhance CAR T-Cell Effectiveness DOI Open Access
Beatriz Coutinho de Oliveira, S. M. Nayeemul Bari, J. Joseph Melenhorst

et al.

Cancers, Journal Year: 2025, Volume and Issue: 17(3), P. 383 - 383

Published: Jan. 24, 2025

Anti-CD19 chimeric antigen receptor (CAR) T-cell therapy represents a breakthrough in the treatment of relapsed and refractory B-cell malignancies, such as chronic lymphocytic leukemia (CLL), inducing long-term, sometimes curative, responses. However, fewer than 30% CLL patients achieve outcomes. It has been shown that smaller subset T cells capable expansion persistence is crucial for effectiveness. Notably, pre-existing mutation epigenetic regulator TET2, combined with CAR vector-induced disruption other intact allele, significantly enhanced potency CAR-engineered clone one patient. This finding aligns independent research, suggesting gene’s genomic insertion site influences tumor-targeting capability. Thus, it plausible gene disruptions affect function. review synthesizes existing knowledge on vector integration into host genome its impact clinical outcomes patients. Our aim to inform development improved therapies enhance their overall efficacy.

Language: Английский

Citations

0