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Effectiveness of SCID-X1 Gene Therapy Influenced by 3D Genome Architecture

Patients with X-linked severe combined immunodeficiency disorder (SCID-X1), often known as “bubble boy disease,” are born with a faulty gene that inhibits immune cells from being produced.

9 Oct 2023

Gene-Silencing Complexes Work Together to Render X Chromosomes Inactive

Two protein complexes are involved in the mysterious process of shutting off one X chromosome in female mammals, according to a new study from RIKEN. This result could aid in the understanding of how specific tumors in women develop.

21 Apr 2023

Study shows how gene related to Nascimento Syndrome activates the cell response to stress

According to a Duke University study, a gene linked to severe learning disabilities in humans also plays a critical role in cells’ response to environmental stress.

31 May 2022

RNA targeted by small molecule drugs, likely to create new pathways for disease treatment

RNA (ribonucleic acid) is involved in many aspects of human health, and a new study published in the journal Nature provides compelling evidence that RNA could be a promising pharmacological target.

1 Apr 2022

Study explores that extra dosages of genes may lead to surplus X chromosomes

One out of every 500 to 1,000 boys is born with one or more extra X chromosomes, which can cause a variety of symptoms as the extra chromosomes to including infertility, larger breasts, osteoporosis, diabetes, cardiac problems, intellectual incapacity, and cancer.

1 Apr 2022

Study reveals mechanism that regulates genes on sex chromosomes

In the human population, males have one X and one Y chromosomes and females have two X chromosomes. As a result, somatic cells have unique mechanisms that maintain the same gene expression levels on the X chromosome between both genders.

10 Mar 2021

Targeting DEL-1 molecule could be an effective way to treat autoimmune disease

Immune response is a balancing act: Too much can lead to inflammatory or autoimmune disease; too little could lead to a serious infection. Regulatory T cells, or Tregs, are important players in striking this balance, acting as "brakes" on the immune response so it doesn't go overboard.

27 Oct 2020

Researchers show how two X chromosomes communicate during embryo development

Researchers at Massachusetts General Hospital (MGH) have solved a mystery that has long puzzled scientists: How do the bodies of female humans and all other mammals decide which of the two X chromosomes it carries in each cell should be active and which one should be silent?

18 Aug 2020

A new cell-based gene therapy approach to correct hemophilia A

In a new study from the Wake Forest Institute for Regenerative Medicine (WFIRM) researchers have developed an optimized cellular platform for delivering Factor 8 to better treat patients with hemophilia A.

29 Jul 2020

New mechanism shows promise for treating certain congenital disorders

Scientists have discovered a major mechanism in the inactivation of X chromosomes, an event that may hold clues to develop treatments for congenital disorders.

16 Jun 2020