Gene Silencing, Gene Therapy and Regulation - Clinical Research Informer

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Gene Silencing

Gene Therapy

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uniQure signs license deal for Apic Bio’s gene therapy for SOD1-ALS

Pharmaceutical Technology

JANUARY 31, 2023

Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.

Gene Therapy

Gene Therapy Licensing Licensing Gene

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

MAY 4, 2021

These modifications regulate gene expression without changing the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies.

DNA

DNA Gene Gene Silencing Genome

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

MAY 4, 2021

These modifications regulate gene expression without altering the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies.

DNA

DNA Gene Gene Silencing Genome

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uniQure signs license deal for Apic Bio’s gene therapy for SOD1-ALS

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

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