Targeting 'horror autotoxicus' by gene therapy: results, challenges and the future

Assembly PCR synthesis of optimally designed, compact, multi-responsive promoters suited to gene therapy application.

Scientific reports·2016

Differences between disease-associated endoplasmic reticulum aminopeptidase 1 (ERAP1) isoforms in cellular expression, interactions with tumour necrosis factor receptor 1 (TNF-R1) and regulation by cytokines.

Clinical and experimental immunology·2014

Gut permeability in IgA nephropathy.

Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association·2013

Historical development of monoclonal antibody therapeutics.

Handbook of experimental pharmacology·2007

Inflammation-inducible anti-TNF gene expression mediated by intra-articular injection of serotype 5 adeno-associated virus reduces arthritis.

The journal of gene medicine·2007

Reduction of arthritis following intra-articular administration of an adeno-associated virus serotype 5 expressing a disease-inducible TNF-blocking agent.

Annals of the rheumatic diseases·2007

An engineered helper plasmid generates differential E4orf6 and L4-22/33K gene expression increasing AAV vector production.

Gene therapy·2026

A gene therapy approach to prevent dilated intercellular space, a hallmark of gastroesophageal reflux disease.

Gene therapy·2026

Precision correction of the GJB2 c.235delC mutation by prime editing in vitro.

Gene therapy·2026

Bone- and muscle-targeted adeno-associated viral vectors enable tissue-selective vitamin D receptor knockdown in mice.

Gene therapy·2026

SACF and GILA for in vitro transformation assessment of CRISPR/Cas9-edited cell therapy candidates: a multi-site study.

Gene therapy·2026

rAAV production cost analysis: Indication-specific cost per dose and reduction strategies.

Gene therapy·2026

Assessment of Natural Killer Cell Serial Degranulation.

Current protocols·2026

Oncogenic KRAS Rewires mRNA Stability and Translation through AGO2-Mediated Disruption of the miRNA pathway.

Cancer research·2026

Stimuli-responsive nanocarriers for precision cation channel therapy.

Chemical communications (Cambridge, England)·2026

Disruption of hippocampal upstream regulators of mTOR and insulin pathways in Down syndrome with Alzheimer's disease neuropathology: Preliminary observations.

Alzheimer's & dementia : the journal of the Alzheimer's Association·2026

ATF7 drives diabetic wound healing via NOTCH1 repression and N1ICD-dependent macrophage polarization control.

JCI insight·2026

Innate immune sensor NOD2 promotes cartilage degradation and osteoarthritis progression by stabilizing TRAF6 in chondrocytes.

JCI insight·2026

Related Experiment Videos

Targeting 'horror autotoxicus' by gene therapy: results, challenges and the future

Related Concept Videos

Assembly PCR synthesis of optimally designed, compact, multi-responsive promoters suited to gene therapy application.

Differences between disease-associated endoplasmic reticulum aminopeptidase 1 (ERAP1) isoforms in cellular expression, interactions with tumour necrosis factor receptor 1 (TNF-R1) and regulation by cytokines.

Gut permeability in IgA nephropathy.

Historical development of monoclonal antibody therapeutics.

Inflammation-inducible anti-TNF gene expression mediated by intra-articular injection of serotype 5 adeno-associated virus reduces arthritis.

Reduction of arthritis following intra-articular administration of an adeno-associated virus serotype 5 expressing a disease-inducible TNF-blocking agent.

An engineered helper plasmid generates differential E4orf6 and L4-22/33K gene expression increasing AAV vector production.

A gene therapy approach to prevent dilated intercellular space, a hallmark of gastroesophageal reflux disease.

Precision correction of the GJB2 c.235delC mutation by prime editing in vitro.

Bone- and muscle-targeted adeno-associated viral vectors enable tissue-selective vitamin D receptor knockdown in mice.

SACF and GILA for in vitro transformation assessment of CRISPR/Cas9-edited cell therapy candidates: a multi-site study.

rAAV production cost analysis: Indication-specific cost per dose and reduction strategies.

Assessment of Natural Killer Cell Serial Degranulation.

Oncogenic KRAS Rewires mRNA Stability and Translation through AGO2-Mediated Disruption of the miRNA pathway.

Stimuli-responsive nanocarriers for precision cation channel therapy.

Disruption of hippocampal upstream regulators of mTOR and insulin pathways in Down syndrome with Alzheimer's disease neuropathology: Preliminary observations.

ATF7 drives diabetic wound healing via NOTCH1 repression and N1ICD-dependent macrophage polarization control.

Innate immune sensor NOD2 promotes cartilage degradation and osteoarthritis progression by stabilizing TRAF6 in chondrocytes.

Related Experiment Videos

Targeting 'horror autotoxicus' by gene therapy: results, challenges and the future

Related Concept Videos

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