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Study reveals molecular machinery that regulates membrane association of Arf proteins

Study reveals molecular machinery that regulates membrane association of Arf proteins

Convalescent plasma in COVID-19 can be effective, especially early in disease

Convalescent plasma in COVID-19 can be effective, especially early in disease

Novel insights open up new avenues for treating autoimmune conditions

Novel insights open up new avenues for treating autoimmune conditions

Distinct DNA methylation signature found in the cord blood of newborns diagnosed with ASD

Distinct DNA methylation signature found in the cord blood of newborns diagnosed with ASD

Researchers find link between dystrophic muscles and lymphatic system in mice with Duchenne disease

Researchers find link between dystrophic muscles and lymphatic system in mice with Duchenne disease

Researchers solve a long-held mystery of X chromosome inactivation

Researchers solve a long-held mystery of X chromosome inactivation

Large-scale analysis of Alport syndrome reveals the effectiveness of existing treatments

Large-scale analysis of Alport syndrome reveals the effectiveness of existing treatments

A new cell-based gene therapy approach to better treat patients with hemophilia A

A new cell-based gene therapy approach to better treat patients with hemophilia A

New editorial published following adverse events of gene therapy clinical trial

New editorial published following adverse events of gene therapy clinical trial

MGH researchers identify key mechanism in X chromosome inactivation

MGH researchers identify key mechanism in X chromosome inactivation

CSL Behring and Seattle Children's partner to develop gene therapies for primary immunodeficiency diseases

CSL Behring and Seattle Children's partner to develop gene therapies for primary immunodeficiency diseases

New study offers clues to why autism is more common in boys than in girls

New study offers clues to why autism is more common in boys than in girls

Model that sheds light on severe Duchenne muscular dystrophy could pave the way for new therapies

Model that sheds light on severe Duchenne muscular dystrophy could pave the way for new therapies

New C. elegans model will accelerate study of a rare disease

New C. elegans model will accelerate study of a rare disease

Wayne State researchers receive grant to develop new treatments for Barth syndrome

Wayne State researchers receive grant to develop new treatments for Barth syndrome

Successful gene therapy trial in patients with X-linked Chronic Septic Granulomatosis

Successful gene therapy trial in patients with X-linked Chronic Septic Granulomatosis

PerkinElmer provides newborn screening assay for PPMD’s New York State pilot program

PerkinElmer provides newborn screening assay for PPMD’s New York State pilot program

Research team publishes findings from study of TAF1 syndrome

Research team publishes findings from study of TAF1 syndrome

Three UCLA researchers receive multimillion-dollar grant from state's stem cell agency

Three UCLA researchers receive multimillion-dollar grant from state's stem cell agency

Researchers make progress toward gene-based strategies to treat inherited neurometabolic diseases

Researchers make progress toward gene-based strategies to treat inherited neurometabolic diseases