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Advances in X chromosome inactivation open therapeutic opportunities for X-linked genetic disorders

A new review explores how advances in understanding X chromosome inactivation (XCI) are creating potential therapeutic opportunities for a range of X-linked genetic disorders, including Rett syndrome, Fabry disease, Duchenne muscular dystrophy, hemophilia, and others.

3 Aug 2026

New AI model improves prediction of cancer immunotherapy success

Cancer immunotherapy drugs known as immune checkpoint inhibitors (ICIs) can be miracle drugs for cancer patients, curing some and turning deadly disease into a manageable chronic condition in others.

3 Jul 2026

Jumping genes help strengthen the mammalian Y chromosome

The humble Y chromosome may be the smallest chromosome in the mammalian genome (and getting even smaller), but it is mighty: genes on the Y chromosome are critical for fertility in males.

1 Jun 2026

New SMArT platform improves safety of CRISPR gene editing

A team of researchers led by Luigi Naldini at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) has developed a new strategy to significantly improve the precision and safety of CRISPR-Cas9 gene editing in human blood stem cells, potentially overcoming one of the major barriers limiting broader clinical application of genome editing therapies.

1 Jun 2026

Scientists identify genetic mechanisms connected to core autism behaviors

A long‑overlooked stretch of the human genome appears to play a distinct role in shaping the social and stereotypic repetitive behaviors that define Autism Spectrum Disorder (ASD), without affecting learning or other cognitive abilities, according to a major new study published in Nature.

13 May 2026

Antisense oligonucleotides reverse rare neurodevelopmental disorder in preclinical models

Scientists at St. Jude Children's Research Hospital have found they can reverse the effects of HNRNPH2-related neurodevelopmental disorder using antisense oligonucleotides (ASOs) in preclinical models. ASOs are short synthetic nucleic acid strands that target specific messenger RNA.

22 Apr 2026

Researchers identify CD99L2 gene as a cause of spastic ataxia

Despite modern high-throughput sequencing, the genetic cause of most rare movement disorders remains unclear.

17 Mar 2026

New method can help better assess precision genome editing technology

Scientists and physicians can better assess precision genome editing technology using a new method made public today by St. Jude Children's Research Hospital. Significant amounts of time and resources spent improving CRISPR gene editing technology focus on identifying small off-target sites that pose a safety risk, which is also technically challenging.

2 Jan 2026

Study highlights need for XDP specific diagnostic assays

Scientists at Brigham and Women's Hospital and Harvard Medical School have developed a targeted genetic test to improve diagnosis for X-linked dystonia-parkinsonism (XDP), a rare and disabling movement disorder that affects primarily men of Filipino ancestry.

17 Nov 2025

Discovery explains why women face higher risk of Alzheimer’s and multiple sclerosis

New research by UCLA Health has identified a sex-chromosome linked gene that drives inflammation in the female brain, offering insight into why women are disproportionately affected by conditions such as Alzheimer's disease and multiple sclerosis as well as offering a potential target for intervention.

15 Oct 2025

New AI model can identify treatments that reverse disease states in cells

In a move that could reshape drug discovery, researchers at Harvard Medical School have designed an artificial intelligence model capable of identifying treatments that reverse disease states in cells.

10 Sep 2025

Researchers uncover a promising new therapeutic target for Barth syndrome

Researchers at The Hospital for Sick Children (SickKids) have uncovered a promising new therapeutic target for Barth syndrome, a rare genetic condition with no current cure.

3 Sep 2025

Virtual memory T cells provide nonspecific immunity for infants early in life

Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune cells, provide nonspecific immunity for infants early in life.

31 Jul 2025

Reactivating silent genes offers hope for Rett syndrome treatment

Researchers led by UC Davis Health scientist Sanchita Bhatnagar have developed a promising gene therapy that could treat Rett syndrome.

23 Jul 2025

Gene therapy shows long-term success in hemophilia B patients

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London today report thirteen years of follow-up data.

12 Jun 2025

New nanopore sequencing assay advances fragile X syndrome carrier screening

Researchers have developed an innovative new nanopore sequencing assay to identify carriers of fragile X syndrome (FXS), the leading cause of monogenic autism spectrum disorder and inherited intellectual disabilities.

12 Jun 2025

Natural compounds from Brazilian plants show promise against stomach cancer

A new review was published in Volume 12 of Oncoscience on May 8, 2025, titled "The chemopreventive effects of native Brazilian plants on stomach cancer: A review of the last 25 years."

22 May 2025

Genetic pathways explain why some people grow tall and others stay short

A landmark review in Nature Reviews Genetics dissects the rare and common genetic variants that shape human height. The paper explains how monogenic and polygenic factors converge on key growth pathways and influence disease risk.

7 Apr 2025

Study reveals the genetic arms race between X and Y sperm

In evolutionary terms, fitness is defined as an organism's ability to survive and reproduce its genes into the next generation.

4 Apr 2025

New study confirms CAR T cell therapy does not cause secondary cancers

Within a large group of more than 700 patients treated with CAR T cell therapy, researchers found no evidence that the therapy itself caused any type of secondary cancer in the modified T cells, according to new analysis reported today in Nature Medicine from the Perelman School of Medicine at the University of Pennsylvania and Penn Medicine's Abramson Cancer Center.

3 Feb 2025