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Home » Topics » Disease categories and therapies » Genetic/congenital

Genetic/congenital
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Cancer cell, DNA illustration
Cancer

New pathway for transcription suggests new target for MYC-driven tumors

July 25, 2023
By Mar de Miguel
The overexpression of the MYC oncogene could be explained through a new pathway that would act before transcription, when MYC binds to DNA. A group of scientists from Spain have identified how the ERK2 kinase interacted with the CDK9 protein, enabling it to bind to DNA in the promoter region of MYC.
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3D illustration of a ribosome constructing messenger RNA molecules
Drug Design, Drug Delivery & Technologies

Even personalized drugs could benefit multiple patients

July 18, 2023
By Mar de Miguel
Using whole genome sequencing, scientists at Boston Children’s Hospital have studied the genes and mutations of ataxia-telangiectasia (A-T) that would respond to treatments with splice-switching antisense oligonucleotides (ASOs). Their work, published on July 12, 2023, in Nature, determined the appropriate individualized genetic therapy for these patients and identified a new drug.
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Pancreas
Endocrine/Metabolic

Genetics explain link between type 2 diabetes and osteoarthritis

July 12, 2023
By Mar de Miguel
Researchers from the Institute of Translational Genomics at Helmholtz Munich have described a genetic overlap between type 2 diabetes (T2D), a disease that is also associated with obesity, and osteoarthritis, a degeneration of the joints that worsens with age and coincides in the factor risk of being overweight. The researchers used genetic data, multiomics and functional analysis of the tissues T2D and osteoarthritis express to identify which genes were associated and correlated with both diseases. They published their results on July 10, 2023, in The American Journal of Human Genetics.
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Red blood cells, DNA
Hematologic

Fulcrum Therapeutics licenses rights from Camp4 to advance novel therapies for Diamond-Blackfan anemia

July 11, 2023
Fulcrum Therapeutics Inc. has entered into a worldwide, exclusive license agreement with Camp4 Therapeutics Corp. to advance the discovery, development and commercialization of new therapeutic agents against an undisclosed target for the potential treatment of Diamond-Blackfan anemia (DBA).
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Sickle cell illustration
Genetic/Congenital

Base editing could be more potent than CRISPR-Cas9 for treating hemoglobinopathies

July 10, 2023
By Helen Albert
Research led by St. Jude Children’s Research Hospital and Harvard University shows base-editing approaches could be more effective than CRISPR-Cas9 gene-editing approaches for treating conditions such as sickle cell disease and β-thalassemia. Writing in the July 3, 2023, issue of Nature Genetics, the researchers compared three base-editing approaches with two CRISPR-Cas9 approaches to increasing levels of fetal hemoglobin in CD34+ hematopoietic stem and progenitor cells, and found one of the base-editing approaches was the most potent.
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Neurology/Psychiatric

Two mutations acquired during development could cause schizophrenia in adulthood

July 7, 2023
By Mar de Miguel
Schizophrenia (SCZ) could be associated with genetic alterations that can appear at the beginning of life. Such somatic variants in the NRXN1 and ABCB11 genes could lead to SCZ, according to researchers at Boston Children’s Hospital.
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3D illustration of chromosomes
Cancer

Some cancers could be addicted to aneuploidy

July 7, 2023
By Mar de Miguel
Alterations in chromosome number can play a role in cancer progression. An analysis of recurrent aneuploidies, such as the duplication of the long arm of chromosome 1, revealed that it was required for the proliferation of cancer cells carrying this alteration, an effect that was similar to so-called oncogene addiction. These findings have therapeutic implications that could benefit cancer patients depending on the genetic singularity of their tumor cells.
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Illustration of prescription pill bottle with DNA on the label.

EAN 2023: Even after breakthroughs, gains need defending

July 5, 2023
By Anette Breindl
At the 2023 Annual Congress of the European Academy of Neurology, Mary Reilly described the relationship between bench and bedside as “a continuous circle of translation,” with each cycle beginning with patients and their needs.
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Illustration of prescription pill bottle with DNA on the label.
Neurology/Psychiatric

EAN 2023: Even after breakthroughs, gains need defending

July 4, 2023
By Anette Breindl
It seems unlikely that American poet and civil rights activist Maya Angelou spent much time thinking about translational research. But two quotes of hers capture the essence of the interplay between bench and bedside: “I did then what I knew how to do. Now that I know better, I do better” and “I’ve learned that I still have a lot to learn.” At the 2023 Annual Congress of the European Academy of Neurology, Mary Reilly described the relationship between bench and bedside as “a continuous circle of translation,” with each cycle beginning with patients and their needs.
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Illustration of motor neuron connecting to muscle fiber
Neurology/Psychiatric

Loss of cyclin D3 function reduces DMD pathogenesis in mdx mouse model

June 29, 2023
Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder affecting roughly 1 in 3,500 males. DMD is due to mutations in the dystrophin gene, which encodes for an exceptionally large 427 kD protein. DMD is characterized by repeated degeneration and regeneration of muscle fibers, but ultimately replacement of muscle with fibrotic and adipose tissue. Despite advances in gene therapy and improvements in quality of life, most patients still die by 30 years of age due to cardiopulmonary failure.
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