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

Journal of Biomedical Science, Год журнала: 2024, Номер 31(1)

Опубликована: Авг. 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.

Язык: Английский

A review on regulatory aspects, challenges and public perception in acceptance of genetically modified foods DOI

A. Kanthi Naveen,

Manmath Sontakke

Food Science and Biotechnology, Год журнала: 2024, Номер 33(4), С. 791 - 804

Опубликована: Янв. 30, 2024

Язык: Английский

Процитировано

8

Prevalence of the CRISPR-cas system and its association with antibiotic resistance in clinical Klebsiella pneumoniae isolates DOI Creative Commons
Hiva Kadkhoda, Pourya Gholizadeh,

Reza Ghotaslou

и другие.

BMC Infectious Diseases, Год журнала: 2024, Номер 24(1)

Опубликована: Июнь 3, 2024

Abstract Background and objective(s) CRISPR-Cas is a prokaryotic adaptive immune system that protects bacteria archaea against mobile genetic elements (MGEs) such as bacteriophages plasmids, transposons. In this study, we aimed to assess the prevalence of systems their association with antibiotic resistance in one most challenging bacterial pathogens, Klebsiella pneumoniae . Materials methods A total 105 K. isolates were collected from various clinical infections. Extended-spectrum β-lactamases (ESBLs) phenotypically detected presence ESBL, aminoglycoside-modifying enzymes (AME), subtype genes identified using PCR. Moreover, diversity was determined by enterobacterial repetitive intergenic consensus (ERIC)-PCR. Results Phenotypically, 41.9% (44/105) found be ESBL producers. significant inverse correlation existed between I-E system’s production isolates. Additionally, frequency bla CTX−M1 (3%), CTX−M9 (12.1%), SHV (51.5%), TEM (33.3%), well some AME aac(3)-Iva (21.2%) ant(2’’)-Ia (3%) significantly lower comparison CRISPR-negative There system. Conclusion The correlated antibiotic-resistant gene (ARGs). had than

Язык: Английский

Процитировано

8

Genes for editing to improve economic traits in aquaculture fish species DOI Creative Commons
Zituo Yang, Guihong Fu,

May Lee

и другие.

Aquaculture and Fisheries, Год журнала: 2024, Номер unknown

Опубликована: Июль 1, 2024

Aquaculture, a critical sector for global food security, faces the challenge of meeting growing demand while protecting wild fish populations. Gene editing, powerful genetic tool, emerges as potential solution. By modifying key genes in fish, it is expected to rapidly enhance growth rate, disease resistance, and other economically important traits, leading increased profitability, sustainability, competitiveness industry. This review navigates complex landscape gene editing aquaculture, exploring successes, challenges, prospects improved productivity, resilience. We provide guidance researchers stakeholders identifying responsible addressing economic, environmental, regulatory, ethical considerations. roadmap paves way future where empowers aquaculture meet needs upholding environmental responsibility.

Язык: Английский

Процитировано

8

CRISPR-Cas gene knockouts to optimize engineered T cells for cancer immunotherapy DOI
Valentine De Castro, Jeanne Galaine, Romain Loyon

и другие.

Cancer Gene Therapy, Год журнала: 2024, Номер 31(8), С. 1124 - 1134

Опубликована: Апрель 12, 2024

Язык: Английский

Процитировано

7

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

Journal of Biomedical Science, Год журнала: 2024, Номер 31(1)

Опубликована: Авг. 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.

Язык: Английский

Процитировано

7