journal
https://read.qxmd.com/read/38767512/aav5-delivery-of-crispr-cas9-mediates-genome-editing-in-the-lungs-of-young-rhesus-monkeys
#1
JOURNAL ARTICLE
Shun-Qing Liang, Andrew Navia, Michelle Ramseier, Xuntao Zhou, Michele L Martinez, Charles C Lee, Chen Zhou, Joae Wu, Jun Xie, Qin Su, Dan Wang, Terence R Flotte, Daniel G Anderson, Alice F Tarantal, Alex Shalek, Guangping Gao, Wen Xue
Genome editing has the potential to treat genetic diseases in a variety of tissues including the lung. We have previously developed and validated a dual adeno-associated virus (AAV) CRISPR platform that supports effective editing in the airways of mice. To validate this delivery vehicle in a large animal model, we have shown that intratracheal instillation of CRISPR/Cas9 in AAV5 can edit a housekeeping gene or a disease-related gene in the lungs of young rhesus monkeys. We observed up to 8% editing of ACE2 in lung lobes after single-dose administration...
May 20, 2024: Human Gene Therapy
https://read.qxmd.com/read/38767504/gene-co-expression-and-mirna-regulation-a-path-to-early-intervention-in-colorectal-cancer
#2
JOURNAL ARTICLE
Jason C Huang, Ming-Chun Li, I-Chieh Huang, Je-Ming Hu, Wei-Zhi Lin, Yu-Tien Chang
Early diagnosis and intervention are pivotal in reducing colorectal cancer (CRC) incidence and enhancing patient outcomes. In this study, we focused on three genes, AQP8, GUCA2B, and SPIB, which exhibit high co-expression and play crucial roles in suppressing early-stage CRC. Our objective was to identify key miRNAs that can mitigate CRC tumorigenesis and modulate the co-expression network involving these genes. We conducted a comprehensive analysis using large-scale tissue mRNA data from The Cancer Genome Atlas (TCGA) and the Gene Expression Omnibus to validate the co-expression of AQP8, GUCA2B, and SPIB, and to assess their diagnostic and prognostic significance in CRC...
May 20, 2024: Human Gene Therapy
https://read.qxmd.com/read/38717950/crispr-cas-genome-editing-in-ex-vivo-human-lungs-to-rewire-the-translational-path-of-genome-targeting-therapeutics
#3
JOURNAL ARTICLE
Kumi Mesaki, Haruchika Yamamoto, Stephen Juvet, Jonathan Yeung, Zehong Guan, Akhi Akhter, Yan Yao, Cameron Dickie, Henna Mangat, Aizhou Wang, Gavin W Wilson, Andrea Mariscal, Jim Hu, Alan R Davidson, Benjamin P Kleinstiver, Marcelo Cypel, Mingyao Liu, Shaf Keshavjee
The ongoing advancements in CRISPR-Cas technologies can significantly accelerate the preclinical development of both in vivo and ex-vivo organ genome-editing therapeutics. One of the promising applications is to genetically modify donor organs prior to implantation. The implantation of optimized donor organs with long-lasting immunomodulatory capacity holds promise for reducing the need for lifelong potent whole-body immunosuppression in recipients However, assessing genome-targeting interventions in a clinically-relevant manner prior to clinical trials remains a major challenge due to the limited modalities available...
May 8, 2024: Human Gene Therapy
https://read.qxmd.com/read/38717948/methodological-validation-of-sedimentation-velocity-analytical-ultracentrifugation-method-for-adeno-associated-virus-and-collaborative-calibration-of-system-suitability-substance
#4
JOURNAL ARTICLE
Xi Qin, Qikun Yu, Xiang Li, Wei Jiang, Xinchang Shi, Wenxiu Hou, Da Zhang, Zhenzhen Cai, Hua Bi, Wenhong Fan, Youxue Ding, Yichen Yang, Biao Dong, Long Chen, DE-Hua Huo, Cong Wang, Yong Zhou, Dening Pei, Miao Ye, Chenggang Liang
Currently, adeno-associated virus (AAV) is one of the primary gene delivery vectors in gene therapy, facilitating long-term in vivo gene expression. Despite being imperative, it is incredibly challenging to precisely assess AAV particle distribution according to the sedimentation coefficient and identify impurities related to capsid structures. This study performed the systematic methodological validation of quantifying the AAV empty and full capsid ratio. This includes specificity, accuracy, precision, linearity, and parameter variables involving the sedimentation velocity analytical ultracentrifugation (SV-AUC) method...
May 8, 2024: Human Gene Therapy
https://read.qxmd.com/read/38661546/intravitreal-delivery-of-raav2-hsyn-hrs1-results-in-retinal-ganglion-cell-specific-gene-expression-and-retinal-improvement-in-the-rs1-ko-mouse
#5
JOURNAL ARTICLE
Yangyang Zheng, Xin Xu, Ruoyue Fan, Haolang Jiang, Qingguo Guo, Xuefei Han, Ying Liu, Guangzuo Luo
X-linked retinoschisis (XLRS) is a monogenic recessive inherited retinal disease which clinically manifests retinal schisis cavities and disproportionate reduction of b-wave amplitude to the a-wave amplitude. Currently there are no approved treatments while the causal agent was identified as the retinoschisin (RS1). In the last decade, gene therapy has got great progress and given hopes to incurable genetic diseases. Preclinical studies demonstrated the treatment benefits of hRS1 gene augmentation therapy in the mouse models...
April 25, 2024: Human Gene Therapy
https://read.qxmd.com/read/38661537/advances-in-bone-targeting-drug-delivery-emerging-strategies-using-adeno-associated-virus
#6
JOURNAL ARTICLE
Tadatoshi Sato, Sachin Chaugule, Matthew Greenblatt, Guangping Gao, Jae-Hyuck Shim
The development of bone-targeting drug delivery systems holds immense promise for improving the treatment of skeletal diseases. By precisely delivering therapeutic agents to the affected areas of bone, these strategies can enhance drug efficacy, minimize off-target effects, and promote patient adherence, ultimately leading to improved treatment outcomes and an enhanced quality of life for patients. This review aims to provide an overview of the current state of affinity-based bone-targeting agents and recent breakthroughs in innovative bone-targeting adeno-associated virus (AAV) strategies to treat skeletal diseases in mice...
April 25, 2024: Human Gene Therapy
https://read.qxmd.com/read/38581431/prevalence-study-of-cellular-capsid-specific-immune-responses-to-aav2-4-5-8-9-and-rh10-in-healthy-donors
#7
JOURNAL ARTICLE
Rebecca Xicluna, Allan Avenel, Céline Vandamme, Marie Devaux, Nicolas Jaulin, Célia Couzinié, Johanne Le Duff, Alicia Charrier, Mickaël Guilbaud, Oumeya Adjali, Gwladys Gernoux
Recombinant adeno-associated virus (rAAV) vectors appear, more than ever, to be efficient viral vectors for in vivo gene transfer as illustrated by the approvals of 7 drugs across Europe and the United States. Nevertheless, preexisting immunity to AAV capsid in humans remains one of the major limits for a successful clinical translation. Whereas a preexisting humoral response to AAV capsid is well documented, the prevalence of preexisting capsid-specific T cell responses still needs to be studied and characterized...
April 19, 2024: Human Gene Therapy
https://read.qxmd.com/read/38450566/role-of-foxp3-regulatory-t-cells-in-modulating-immune-responses-to-adeno-associated-virus-gene-therapy
#8
JOURNAL ARTICLE
Maite Muñoz-Melero, Moanaro Biswas
Adeno-associated virus (AAV) gene therapy is making rapid strides owing to its wide range of therapeutic applications. However, development of serious immune responses to the capsid antigen or the therapeutic transgene product hinders its full clinical impact. Immune suppressive (IS) drug treatments have been used in various clinical trials to prevent the deleterious effects of cytotoxic T cells to the viral vector or transgene, although there is no consensus on the best treatment regimen, dosage, or schedule...
April 11, 2024: Human Gene Therapy
https://read.qxmd.com/read/38534217/development-of-aav-mediated-gene-therapy-approaches-to-treat-skeletal-diseases
#9
JOURNAL ARTICLE
Chujiao Lin, Matthew B Greenblatt, Guangping Gao, Jae-Hyuck Shim
Adeno-associated viral (AAV) vectors have emerged as crucial tools in advancing gene therapy for skeletal diseases, offering the potential for sustained expression with low postinfection immunogenicity and pathogenicity. Preclinical studies support both the therapeutic efficacy and safety of these vectors, illustrating the promise of AAV-mediated gene therapy. Emerging technologies and innovations in AAV-mediated gene therapy strategies, such as gene addition, gene replacement, gene silencing, and gene editing, offer new approaches to clinical application...
April 8, 2024: Human Gene Therapy
https://read.qxmd.com/read/38527236/-correction-to-efficacy-and-safety-of-adeno-associated-virus-based-clinical-gene-therapy-for-hemophilia-a-systematic-review-and-meta-analysis-by-han-et-al-hum-gene-ther-2024-35-3-4-93-103-doi-10-1089-hum-2023-208
#10
JOURNAL ARTICLE
https://read.qxmd.com/read/38526393/a-review-of-the-cost-effectiveness-evidence-for-fda-approved-cell-and-gene-therapies
#11
JOURNAL ARTICLE
Sumaya Abuloha, Shu Niu, Darlene Adirika, Benjamin P Harvey, Mikael Svensson
Cell and gene therapy innovations have provided several significant breakthroughs in recent years. However, cell and gene therapies often come with a high upfront cost, raising questions about patient access, affordability, and long-term value. This study reviewed cost-effectiveness analysis studies that have attempted to assess the long-term value of FDA-approved cell and gene therapies. Two reviewers independently searched the Tufts Medical Center Cost-Effectiveness Analysis Registry to identify all studies for FDA-approved cell and gene therapies per January 2023...
March 25, 2024: Human Gene Therapy
https://read.qxmd.com/read/38420654/pre-existing-immunity-to-a-nucleic-acid-contaminant-derived-antigen-mediates-transaminitis-and-resultant-diminished-transgene-expression-in-a-mouse-model-of-hepatic-raav-mediated-gene-transfer
#12
JOURNAL ARTICLE
Mark Brimble, Christopher L Morton, Stephen M Winston, Isaiah L Reeves, Yunyu Spence, Pei-Hsin Cheng, Jungfang Zhou, Amit C Nathwani, Paul Glwndyr Thomas, Aisha Souquette, Andrew M Davidoff
Liver injury with concomitant loss of therapeutic transgene expression can be a clinical sequela of systemic administration of rAAV when used for gene therapy, and a significant barrier to treatment efficacy. Despite this, it has been difficult to replicate this phenotype in preclinical models, thereby limiting the field's ability to systematically investigate underlying biological mechanisms and develop interventions. Prior animal models have focused on capsid and transgene-related immunogenicity but the impact of concurrently present non-transgene or vector antigens on therapeutic efficacy, such as those derived from contaminating nucleic acids within rAAV preps, has yet to be investigated...
February 29, 2024: Human Gene Therapy
https://read.qxmd.com/read/38781422/verve-pauses-enrollment-in-base-editing-trial-after-adverse-events
#13
JOURNAL ARTICLE
Alex Philippidis
No abstract text is available yet for this article.
May 2024: Human Gene Therapy
https://read.qxmd.com/read/38626340/remembrances-of-kenneth-berns-phd
#14
JOURNAL ARTICLE
(no author information available yet)
No abstract text is available yet for this article.
April 2024: Human Gene Therapy
https://read.qxmd.com/read/38626339/orchard-therapeutics-gains-first-u-s-approval-for-a-metachromatic-leukodystrophy-gene-therapy
#15
JOURNAL ARTICLE
Alex Philippidis
No abstract text is available yet for this article.
April 2024: Human Gene Therapy
https://read.qxmd.com/read/38323580/the-new-frontiers-of-gene-therapy-and-gene-editing-in-inflammatory-diseases
#16
REVIEW
Alessandro Romano, Alessandra Mortellaro
Inflammatory diseases are conditions characterized by abnormal and often excessive immune responses, leading to tissue and organ inflammation. The complexity of these disorders arises from the intricate interplay of genetic factors and immune responses, which challenges conventional therapeutic approaches. However, the field of genetic manipulation has sparked unprecedented optimism in addressing these complex disorders. This review aims to comprehensively explore the application of gene therapy and gene editing in the context of inflammatory diseases, offering solutions that range from correcting genetic defects to precise immune modulation...
April 2024: Human Gene Therapy
https://read.qxmd.com/read/38497910/kenneth-i-berns-md-phd-1938-2024
#17
JOURNAL ARTICLE
Barry J Byrne, Terence R Flotte, Roland W Herzog, Arun Srivastava
No abstract text is available yet for this article.
March 2024: Human Gene Therapy
https://read.qxmd.com/read/38497909/akouos-therapy-restores-11-year-old-boy-s-hearing-after-1-month
#18
JOURNAL ARTICLE
Alex Philippidis
No abstract text is available yet for this article.
March 2024: Human Gene Therapy
https://read.qxmd.com/read/38386514/combination-immunotherapy-of-oncolytic-flu-vectored-virus-and-programmed-cell-death-1-blockade-enhances-antitumor-activity-in-hepatocellular-carcinoma
#19
JOURNAL ARTICLE
Hongyu Yu, Fang Sun, Yan Xu, Hao Yang, Chongyu Tian, Cong Li, Yimin Kang, Lei Hao, Penghui Yang
Oncolytic viruses (OVs) are appealing anti-tumor agents. But it is limited in its effectiveness. In this study, we used combination therapy with immune checkpoint inhibitor to enhance the antitumor efficacy of OVs. Using reverse genetics technology, we rescued an oncolytic influenza virus with the name delNS1-GM-CSF from the virus. After identifying the hemagglutination and 50% tissue culture infectivedose (TCID50 ) of delNS1-GM-CSF, it was purified, and the viral morphology was observed under electron microscopy...
March 2024: Human Gene Therapy
https://read.qxmd.com/read/38386502/current-status-and-prospects-of-viral-vector-based-gene-therapy-to-treat-kidney-diseases
#20
REVIEW
Louise Medaer, Koenraad Veys, Rik Gijsbers
Inherited kidney diseases are among the leading causes of chronic kidney disease, reducing the quality of life and resulting in substantial socioeconomic impact. The advent of early genetic testing and the growing understanding of the molecular basis and pathophysiology of these disorders have opened avenues for novel treatment strategies. Viral vector-based gene therapies have evolved from experimental treatments for rare diseases to potent platforms that carry the intrinsic potential to provide a cure with a single application...
March 2024: Human Gene Therapy
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