A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly developed form of CRISPR at UNSW Sydney points to a safer way of treating inherited conditions such as Sickle Cell disease.

Gene editing has moved from theory to bedside with a speed that would have seemed impossible a decade ago. A new wave of CRISPR advances is not only correcting single mutations in the lab but beginning to reshape how doctors think about treating inherited disease in living patients.

Rare genetic disorders are typically caused by a variant in a single gene. It is estimated that there are over 7000 such conditions, meaning that although individually rare, they are collectively common. Understanding the genetic basis of a rare disorder ...

Degenerative diseases are a group of conditions characterized by the progressive deterioration of cells, tissues and organs. While some degenerative diseases arise due to environmental factors or lifestyle choices, others have a strong hereditary component.

Abu Dhabi successfully completes its first gene therapy for genetic blood disorders, revolutionizing treatment approaches for sickle cell disease and beta thalassemia. Discover the implications of this groundbreaking achievement in UAE healthcare.

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life. Tomorrow, we will intervene before birth. For the first ...

When a child inherits a genetic condition, it often means both parents unknowingly carried genetic variations that, when combined, led to the disease. Modern genetic testing now allows families to understand their risk factors better, though many face ...

Genetic testing can help doctors diagnose some neurological disorders, such as Huntington’s disease. It can also provide insights into a person’s risk of future health conditions. Genetic testing allows for the early identification of hereditary ...

A recent study finds that certain psychiatric disorders have considerable genetic overlap. But these genes aren’t the whole story; you have a say in what they do.

Inherited platelet disorders represent a heterogeneous group of genetic conditions that affect the number, size, or function of platelets, with clinical manifestations ranging from mild mucocutaneous bleeding to life‐threatening haemorrhage. Recent ...