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Researchers reveal molecular cause for severe multi-organ disorder

Researchers reveal molecular cause for severe multi-organ disorder

New drug provides hope for patients with Duchenne muscular dystrophy

New drug provides hope for patients with Duchenne muscular dystrophy

Study demonstrates viability of gene editing strategy for recessive dystrophic epidermolysis bullosa

Study demonstrates viability of gene editing strategy for recessive dystrophic epidermolysis bullosa

Researchers develop new method to boost efficiency of CRISPR gene editing in DMD

Researchers develop new method to boost efficiency of CRISPR gene editing in DMD

Single CRISPR treatment can safely and stably correct genetic disease

Single CRISPR treatment can safely and stably correct genetic disease

Study points to potential new biomarker and drug target for amyotrophic lateral sclerosis

Study points to potential new biomarker and drug target for amyotrophic lateral sclerosis

Research may pave way for development of potent weapons against cancer

Research may pave way for development of potent weapons against cancer

Gene editing could be used to halt kidney disease in patients with Joubert syndrome

Gene editing could be used to halt kidney disease in patients with Joubert syndrome

Study: Targeting specific genomic mutation in breast cancer improves survival

Study: Targeting specific genomic mutation in breast cancer improves survival

Deciphera presents Phase 1 clinical results of DCC-2618 in patients with gastrointestinal stromal tumors

Deciphera presents Phase 1 clinical results of DCC-2618 in patients with gastrointestinal stromal tumors

New blood test may detect early-stage lung cancer when surgical intervention is not possible

New blood test may detect early-stage lung cancer when surgical intervention is not possible

Pfizer announces FDA approval of VIZIMPRO for first-line treatment of EGFR-mutated metastatic NSCLC

Pfizer announces FDA approval of VIZIMPRO for first-line treatment of EGFR-mutated metastatic NSCLC

Urine exRNA may be source of biomarkers for muscular dystrophy

Urine exRNA may be source of biomarkers for muscular dystrophy

New assay may help improve both sarcoma diagnosis and treatment

New assay may help improve both sarcoma diagnosis and treatment

New CRISPR technology may help eliminate mutated gene sequence

New CRISPR technology may help eliminate mutated gene sequence

Hearing partially restored in deaf lab mice using novel therapy

Hearing partially restored in deaf lab mice using novel therapy

KFU medical departments seek to find treatments for hereditary syndromes

KFU medical departments seek to find treatments for hereditary syndromes

New guidelines intend to provide template for optimal, most up-to-date DMD care

New guidelines intend to provide template for optimal, most up-to-date DMD care

Human sperm may hold potential to serve as biomarkers of future health

Human sperm may hold potential to serve as biomarkers of future health

UT Southwestern researchers develop improved gene-editing technique to correct DMD mutations

UT Southwestern researchers develop improved gene-editing technique to correct DMD mutations