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Adeno-associated virus: The gene therapy revolution faces manufacturing and safety hurdles

Recombinant adeno-associated virus (rAAV)-based genetic applications to treat human diseases.

5 Apr 2024

Study reveals the inner workings of gene mutations linked to ultra-rare syndrome

A team from the University of Ottawa's Faculty of Medicine has completed an exciting new study that reveals the inner workings of gene mutations that result in an ultra-rare syndrome with fewer than 100 reported cases since its first description in the early 1960s.

18 Mar 2024

Females' immune system edges out males but at a cost of higher autoimmune risk

A review article highlights how two X chromosomes in females influence immune system composition and function, leading to faster infection clearance and stronger vaccination responses but also a higher risk of autoimmune diseases.

22 Feb 2024

Xist lncRNA identified as key trigger in female-biased autoimmune diseases

Researchers uncover that the Xist ribonucleoprotein complex, unique to females, is a key driver of sex-biased autoimmunity, offering new insights into autoimmune disorders' higher prevalence in women.

4 Feb 2024

RBMX protein emerges as key player in cancer progression and immunotherapy response

A new research paper was published in Aging (listed by MEDLINE/PubMed as "Aging (Albany NY)" and "Aging-US" by Web of Science) Volume 16, Issue 1, entitled, "Aberrant RBMX expression is relevant for cancer prognosis and immunotherapy response."

17 Jan 2024

Researchers report progress in the search for more accurate blood markers for acute kidney injury

Using cells from kidney biopsies, Johns Hopkins Medicine, researchers report progress in the search for more accurate and easier-to-obtain markers to help predict, manage and assess treatment of acute kidney injury (AKI).

14 Dec 2023

Key gene linked to male bias in autism, Tourette's, and ADHD uncovered

Sex bias in attention deficit hyperactivity disorder (ADHD), autism spectrum disorder (ASD), and Tourette syndrome (TS).

8 Dec 2023

JAK inhibitors and tocilizumab elicit better response rates in VEXAS syndrome

New research at ACR Convergence 2023, the American College of Rheumatology's (ACR) annual meeting, found that JAK inhibitors (JAKi) and tocilizumab elicited better response rates in VEXAS syndrome compared to other targeted therapies.

7 Nov 2023

Deep brain stimulation improves memory and promotes neuron growth in Rett mice

A recent study from the lab of Dr. Jianrong Tang, associate professor at Baylor College of Medicine and principal investigator at the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children's Hospital shows that deep brain stimulation (DBS) of a specific brain region results in a significant and sustained improvement of memory in Rett mice.

13 Oct 2023

Differences in immune responses to IL-6 inhibition therapy between male and female COVID-19 patients

Researchers investigated whether anti‑interleukin 6 monoclonal antibodies were related to heterogenous treatment effects between male and female COVID-19 patients.

24 Aug 2023

Nationwide Children's Hospital pioneers new gene therapy for Duchenne muscular dystrophy

In a landmark moment for the Abigail Wexner Research Institute at Nationwide Children's, a 5-year-old from Bellefontaine, Ohio, received the first dose of a recently approved gene therapy for Duchenne muscular dystrophy at Nationwide Children's Hospital, where the therapy was invented and initially tested.

8 Aug 2023

Study sheds light on mechanisms behind a rare disease found almost exclusively in boys

A University of Ottawa-led research team has published rigorous new research that advances a quest to understand a puzzling – and heartbreaking – ultra-rare disease that's found almost exclusively in boys.

3 Aug 2023

New porcine model could help develop treatments for Dduchenne muscular dystrophy

The group of LMU scientist Eckhard Wolf has developed a porcine DMD model with a mutation, which mimics the hallmarks of the human disease but develops them in an accelerated mode.

12 Jul 2023

Decreased viral infection severity in females may be due to extra copy of X chromosome-linked gene

It has long been known that viral infections can be more severe in males than females, but the question as to why has remained a mystery – until possibly now.

17 Mar 2023

Individuals with autosomal and X-linked recessive deficiency of IRAK-4 and MyD88 are more susceptible to developing hypoxemic COVID-19 pneumonia

Researchers identified that individuals with a deficiency of myeloid differentiation primary response 88 (MyD88) and interleukin-1 receptor-associated kinase 4 (IRAK-4) due to an autosomal recessive condition were highly susceptible to developing hypoxemic coronavirus disease 2019 (COVID-19) pneumonia when infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2).

9 Mar 2023

New research may explain why some patients suffer from long-COVID

A review article published in The Journal of Clinical Investigation deciphers the relationship between rare inborn errors of type I interferon immunity and the development of severe coronavirus disease 2019 (COVID-19) pneumonia.

6 Feb 2023

Study provides an estimate of people in the United States with VEXAS syndrome

About 13,200 men and another 2,300 women in the United States over age 50 are estimated to have VEXAS syndrome, according to a new study led by researchers at NYU Grossman School of Medicine.

24 Jan 2023

Researchers discover previously unknown mechanism for how 'starvation response' is triggered

Body cells burn off fat reserves when nutrient supply from food ceases. A team led by Professor Volker Haucke and Dr. Wonyul Jang from the Leibniz-Forschungsinstitut für Molekulare Pharmakologie (FMP) has now discovered a previously unknown mechanism for how this “starvation response” is triggered, and what can inhibit it.

15 Dec 2022

Gene may play a broader role in embryonic organ development

An international study led by the medical Faculty of the University of Bonn has identified a gene that plays an important role in the development of the human embryo. If it is altered, malformations of various organ systems can result.

15 Nov 2022

Rare variants of type I IFN immunity genes could underlie life-threatening COVID-19

Researchers assessed whether loss-of-function variants of type I interferon immunity genes are associated with life-threatening COVID-19.

27 Oct 2022