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Gene therapy could save children from a rare genetic disease

This impressive achievement could potentially revolutionize how we treat cancer and immunity deficiencies.

Children born with Artemis-SCID face many challenges, from a missing repertoire of T and B cells to reduced resistance against chemotherapy used in bone marrow transplants. Additionally, malfunctioning DNA repair mechanisms increase the risk of developing graft-versus-host disease, where the donor’s immune system attacks host tissues.

That’s why researchers are trying everything to find an antidote for such a rare genetic disease and have now turned to gene therapy to treat Artemis-SCID. Gene therapy eliminates the need for donor cells.


Infant gene therapy – a breakthrough to save Artemis-SCID children

In a recent medical breakthrough, scientists have discovered how to use gene therapy to treat babies born with Severe Combined Immunodeficiency (SCID), or “bubble boy syndrome,” without needing immune-suppressing drugs.

This new innovation has proven to be potentially life-changing for infants suffering from rare diseases, giving them an exponentially improved chance of leading a relatively healthy and normal life.

Scientists break new ground in cancer fight by ‘reinvigorating’ T cells

A new study develops ‘environment-modulating’ drugs that reinvigorate T-cells to once again destroy cancer cells.

T-cells, typically thought to be anti-cancer, can switch sides and work against us in the right environment (or battlefield!), according to a new study published on December 21 in Nature.

This discovery is unexpected because many extensive studies before this believed that most worn-out T-cells’ properties were “irreversible.” Simply put, they were doomed to being subpar tumor killers.


Meletios Verras/iStock.

In light of this, the study went a step further by proving that altering the local environment around the tumor could “reinvigorate” T-cells to once again destroy cancer cells.

Shutting down a mysterious gene can help patients get rid of the deadliest brain cancer

The deadly brain cancer is no longer a mystery.

Glioblastoma is one of the most dangerous cancer types affecting the human brain and spinal cord. Over 240,000 people lose their lives because of nervous system cancer annually, and in most of these cases, the leading cause of death is glioblastoma.

Its tumors spread fast and induce highly painful seizures and headaches. What’s worse is that there is no known 100 percent effective cure for this disease. US president Joe Biden’s eldest son Beau Biden and late American actor Robert Forster were also among the many victims of glioblastoma.

Robust Mouse Rejuvenation project details announced

The newly-created Longevity Escape Velocity Foundation (LEV) has released details of the first study in its flagship research programme: Robust Mouse Rejuvenation – Study 1.

Longevity. Technology: A highlight of Longevity Summit Dublin 2022 was Dr Aubrey de Grey’s announcement of his new foundation; LEV Foundation exists to proactively identify and address the most challenging obstacles on the path to the widespread availability of genuinely effective treatments to prevent and reverse human age-related disease, and to that end, its flagship research programme is a sequence of large mouse lifespan studies.

Mouse models are significant in aging research for several reasons. Mice and humans share many genetic and physiological similarities, including similar aging-related pathways, and this makes mice a useful model for studying the molecular and cellular processes underlying aging in humans.

Big dynorphin may protect neurons from the accumulation of Alzheimer’s-associated amyloid

𝐀𝐥𝐳𝐡𝐞𝐢𝐦𝐞𝐫’𝐬 𝐃𝐢𝐬𝐞𝐚𝐬𝐞


One of the main features of Alzheimer’s disease is that the β-amyloid peptide, a molecule found inside neurons that has many diverse functions, begins to fold incorrectly and accumulates. This process, which ends up causing neuronal death, is linked to a series of other cellular alterations, making it difficult to determine whether they are the cause or the consequence. An example is the case of the deregulation of a type of dynorphin.

Dynorphins are the body’s own opioid peptides, which play a key role in many brain pathways. They are located in different areas of the brain, such as the hippocampus, amygdala or hypothalamus, and are involved in memory processes, emotion control, stress and pain, and among other processes. In addition, several studies have shown their involvement in epilepsy, stroke, addictions, depression and schizophrenia.

Now, in a study published in the Computational and Structural Biotechnology Journal, a research group led by Àlex Perálvarez-Marín, researcher in the Department of Biochemistry and Molecular Biology and the UAB Institut de Neurociències, has studied from computer models and which interactions may exist between β-amyloid peptide and big dynorphin, to determine its role in β-amyloid accumulation.

Gravitas: Wuhan Virus returns: Is India prepared?

The Chinese subvariant Omicron BF.7 has reached India. 3 cases have been reported so far, 2 in Gujarat & 1 in Odisha. The Niti Aayog says there’s no need to panic. But if the situation escalates, Is India prepared? Here’s what Priyanka Sharma has to say.
#china #covid #wion.

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FDA weighs new guidance about risks of LASIK eye surgery

Laser technician Denise Sediq prepares the right eye of Lt. Colonel Thomas Hite, Jr. for corrective eye surgery at Walter Reed Army Medical Center June 11, 2002 in Washington, DC. (Photo by Stefan Zaklin/Getty Images)

The federal agency has issued a 25-page draft report outlining how doctors should better inform patients about the potential for double vision, dry eyes, poor night vision, eye pain and other side effects after LASIK surgery.

A breakthrough in mRNA therapies could lead to biological heart pacemakers

“This concept that cells ‘fight back’ against modified RNA is of practical importance, as it suggests how one might improve the effectiveness of RNA therapy.”

Researchers from the Smidt Heart Institute have unveiled a novel concept — they harnessed modified messenger RNA (mRNA) technology used in creating the Pfizer and Moderna Covid-19 vaccines, which can be a significant step in the evolution and creation of biological pacemakers.


Luismmolina/iStock.

The investigators identified how biological pacemaker cells could “fight back” against therapies to biologically correct abnormal heartbeat rates. They’ve also found a new way to “boost the effectiveness” of RNA therapies by controlling the “fighting back” activity, they said in a release.

Circulating Tumor Cells Effectively Inform Treatment Decisions in Patients with Metastatic Breast Cancer

Circulating tumor cells are tumor cells found circulating in the blood that come from a primary tumor. Circulating tumor cells have utility in clinical applications, including genomic analysis of cancer cells and monitoring the progression or recurrence of cancer. Since circulating tumor cells are usually collected through a blood sample, this method is relatively noninvasive compared with traditional tissue biopsies.

Research presented at the San Antonio Breast Cancer Symposium (held December 6–10, 2022) suggests that a count of circulating tumor cells could help clinicians decide between chemotherapy and endocrine therapy as first-line treatment for certain types of breast cancer.

Using circulating tumor cell (CTC) count to decide between first-line treatments provided an overall survival benefit for patients with metastatic, estrogen receptor (ER)-positive/HER2-negative breast cancer when compared to the physician’s choice of treatment.