Opportunities and challenges in targeting genes for therapy

Caspases shutdown nonsense-mediated mRNA decay during apoptosis.

Cell death and differentiation·2015

Generation of SV40-transformed rabbit tracheal-epithelial-cell-derived blastocyst by somatic cell nuclear transfer.

Cell and tissue research·2012

Bactofection of lung epithelial cells in vitro and in vivo using a genetically modified Escherichia coli.

Gene therapy·2008

Established cell lines used in cystic fibrosis research.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society·2004

Isolation of CF cell lines corrected at DeltaF508-CFTR locus by SFHR-mediated targeting.

Gene therapy·2002

Application of SFHR to gene therapy of monogenic disorders.

Gene therapy·2002

FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome.

Gene therapy·2026

Improving the precision of AAV lung gene therapy for SP-B deficiency using computationally derived lung-specific promoters.

Gene therapy·2026

Recent advancements in improving cross-species applicability of bioengineered AAV capsids.

Gene therapy·2026

Assessment of F/HN-pseudotyped lentiviral vector following intravenous delivery to mice.

Gene therapy·2026

Applications of genome editing technologies in the treatment of human diseases.

Gene therapy·2026

High resolution ES-DMA quantifies AAV capsid DNA content by electrical mobility to mass correlation.

Gene therapy·2026

Development of a Novel, Eco-Friendly Mechanical Cell Lysis Process Using Hollow Fiber Filter for AAV Manufacturing.

Biotechnology and bioengineering·2026

Programmed repair of disease-causing UGA premature termination codons in mammalian brain.

Nucleic acids research·2026

Case Report: Novel homozygous pathogenic variant of the <i>SPG20</i> gene causes the Troyer syndrome in China.

Frontiers in genetics·2026

Molecular profiling and functional analyses of umbilical cord mesenchymal stem cell-derived extracellular vesicles for dry eye diseases.

Extracellular vesicles and circulating nucleic acids·2026

Immunotherapy for tuberculosis: current landscape, mechanistic insights, and translational perspectives.

Frontiers in immunology·2026

Real-World Effectiveness and Safety of Tildrakizumab in Patients With Moderate-to-Severe Psoriasis After Failure of Ustekinumab: A Multicenter Retrospective Study.

The Journal of clinical and aesthetic dermatology·2026

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Opportunities and challenges in targeting genes for therapy

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Caspases shutdown nonsense-mediated mRNA decay during apoptosis.

Generation of SV40-transformed rabbit tracheal-epithelial-cell-derived blastocyst by somatic cell nuclear transfer.

Bactofection of lung epithelial cells in vitro and in vivo using a genetically modified Escherichia coli.

Established cell lines used in cystic fibrosis research.

Isolation of CF cell lines corrected at DeltaF508-CFTR locus by SFHR-mediated targeting.

Application of SFHR to gene therapy of monogenic disorders.

FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome.

Improving the precision of AAV lung gene therapy for SP-B deficiency using computationally derived lung-specific promoters.

Recent advancements in improving cross-species applicability of bioengineered AAV capsids.

Assessment of F/HN-pseudotyped lentiviral vector following intravenous delivery to mice.

Applications of genome editing technologies in the treatment of human diseases.

High resolution ES-DMA quantifies AAV capsid DNA content by electrical mobility to mass correlation.

Development of a Novel, Eco-Friendly Mechanical Cell Lysis Process Using Hollow Fiber Filter for AAV Manufacturing.

Programmed repair of disease-causing UGA premature termination codons in mammalian brain.

Case Report: Novel homozygous pathogenic variant of the <i>SPG20</i> gene causes the Troyer syndrome in China.

Molecular profiling and functional analyses of umbilical cord mesenchymal stem cell-derived extracellular vesicles for dry eye diseases.

Immunotherapy for tuberculosis: current landscape, mechanistic insights, and translational perspectives.

Real-World Effectiveness and Safety of Tildrakizumab in Patients With Moderate-to-Severe Psoriasis After Failure of Ustekinumab: A Multicenter Retrospective Study.

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Opportunities and challenges in targeting genes for therapy

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