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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

Researchers discover genes linked to sex ratio and male fertility in mice

Researchers discover genes linked to sex ratio and male fertility in mice

Researchers identify gene mutation that causes musculoskeletal problems in children

Researchers identify gene mutation that causes musculoskeletal problems in children

Cure for Rett Syndrome a step closer as scientists successfully reactivate 'back-up’ genes

Cure for Rett Syndrome a step closer as scientists successfully reactivate 'back-up’ genes

Disease-modifying therapy for Alport syndrome still remains an unmet need

Disease-modifying therapy for Alport syndrome still remains an unmet need

Rettsyndrome.org funds research that will benefit boys and girls with MECP2-related disorders

Rettsyndrome.org funds research that will benefit boys and girls with MECP2-related disorders

New drug could alleviate symptoms of rare musculoskeletal condition

New drug could alleviate symptoms of rare musculoskeletal condition